Download CV - Toretsky Lab - Georgetown University

Curriculum Vitae
Jeffrey Alan Toretsky, M.D.
Professor with Tenure
Departments of Oncology and Pediatrics
Georgetown University
April 8, 2016
Personal Information
Business Address:
3970 Reservoir Rd N.W.
Room 311 West
Washington, D.C. 20057-1469
Office number: 202-687-8655
Fax number: 202-687-1434
[email protected]
Home Address:
9510 Clement Rd.
Silver Spring, MD 20910
301-588-4444
Place and Date of Birth:
November 8, 1961, Minneapolis, MN
No fluent foreign languages
Licensure
State of Maryland License Number D42381, initially licensed 9/94; inactive 9/30/11
District of Columbia Medical License MD33730, expires 12/31/16
Certifications
American Board of Pediatrics, General Pediatrics, 1991 - 2005
American Board of Pediatrics, Hematology/Oncology, 1994, renewed 2001, 2009
Education
1984 B.S. with honors Biochemistry University of Wisconsin, Madison, WI
1988 M.D.
Medicine
University of Minnesota, Minneapolis, MN
Post Graduate Training
Internship:
1988- 1989, Duke University Medical Center, Department of Pediatrics, Durham, NC,
Chairman Samuel Katz.
Residency:
1989- 1991, Medical College of Virginia, Department of Pediatrics, Richmond, VA
Chairman Harold Mauer
Pediatric Oncology Fellowship:
1991 - 1994, National Cancer Institute, Pediatric Branch, Bethesda, MD
Chief Philip Pizzo
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Post-Fellowship Training:
1994 - 1995, National Cancer Institute, Clinical Pharmacology Branch, Bethesda, MD,
Supervisor Len Neckers
1995 - 1997, National Cancer Institute, Pediatric Branch, Bethesda, MD,
Supervisor Lee Helman
Professional Experience
1997 – 1999, Special Volunteer, Pediatric Oncology Branch, National Cancer Institute,
NIH, Bethesda, MD 20892
1997 – 2002, Assistant Professor, Department of Pediatrics, University of Maryland,
Baltimore. Baltimore, MD
1997 – 2002, Assistant Professor, Program in Oncology, Greenebaum Cancer Center,
University of Maryland, Baltimore. Baltimore, MD
1997 – 2002, Assistant Professor, Program in Molecular and Cell Biology, University of
Maryland, Baltimore. Baltimore, MD
2001 – 2002, Assistant Professor, Department of Biochemistry and Molecular Biology,
University of Maryland, Baltimore. Baltimore, MD
2002 – 2005, Assistant Professor, Department of Oncology, Lombardi Comprehensive
Cancer Center, Georgetown University, Washington, DC
2002 – 2005, Assistant Professor, Department of Pediatrics, Georgetown University,
Washington, DC
2005 – 2011, Associate Professor with tenure, Department of Oncology, Lombardi
Comprehensive Cancer Center, Georgetown University, Washington, DC
2005 – 2011, Associate Professor with tenure, Department of Pediatrics, Georgetown
University, Washington, DC
2011 – present, Professor with tenure, Department of Oncology, Lombardi
Comprehensive Cancer Center, Georgetown University, Washington, DC
2011 – present, Professor with tenure, Department of Pediatrics, Georgetown
University, Washington, DC
2012 – present, Co-director Children’s National Medical Center Multi-disciplinary
Sarcoma Clinic, Washington, DC
Biotechnology/Pharmaceutical Experience
2010 - 2013 Founder, TDP Biotherapeutics, Inc.
This start-up obtained grant funding to license and develop YK-4-279
(USPTO granted to Georgetown University, see patents, below) as a
targeted therapy against EWS-FLI1 of Ewing sarcoma.
2014 - present Founder, Tokalas, Inc. Currently a consultant.
This start-up relicensed YK-4-279 (USPTO granted to Georgetown
University, see patents, below) and successfully completed Series A and
Series B financing in order to complete a chemistry program, toxicology,
pharmacokinetics, and manufacturing. Investigational New Drug
application discussion underway with U.S. FDA and plans to file IND 4th
quarter 2015. Clinical trial sites selected and planning underway for
clinical trial to begin 1st quarter 2016.
Honors and Awards
Outstanding Faculty Entrepreneur in Medical Science, Georgetown Business School,
2012
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Georgetown University Medical Center Convocation Honoree for Outstanding
Research, 2009
Marquis Who’s Who in America Biography, 2009
National Academies Keck Futures Initiatives Conference, 2009
Georgetown University Medical Center Convocation Honoree, 2008
Burroughs Wellcome Clinical Scientist Award in Translational Research, 2008
Connective Tissue Oncology Society Best Pediatric Oncology Poster, 2007
American Society of Clinical Investigation Member, 2007
International Society of Pediatric Oncology Fasanelli Prize, 1999
American Society of Clinical Oncology Young Investigator Award, 1998
National Institutes of Health Fellows Award for Research Excellence, 1997
American Association of Cancer Research Travel Award, 1997
American Society of Pediatric Hematology/Oncology Young Investigator Award, 1996
Max Seham Community Pediatrics Award, 1988
Professional Societies
Biophysical Society, Intrinsically Disordered Protein section, 2011 - present
American Association for Cancer Research, 1996 – present
American Society of Pediatric Hematology and Oncology, 1997 – present
Connective Tissue Oncology Society, 2007 – present
American Association for the Advancement of Science, 1998 – 2006
American Society of Clinical Oncology, 1998 – 2000; 2012 – present
American Academy of Pediatrics, 1991 – 2003
Endocrine Society, 1998 – 2003
IGF Society, 1999 – 2010
Public Service
Local Service
American Cancer Society Intramural Grant Review Committee, 2000 – present
Dr. Arthur Schwartz Award, reviewer 2001
National Service
Grant Review
American Cancer Society study section: Tumor Biology and Endocrinology full
member, January 2003 – 2007
NIH CSR special emphasis panel: National Cooperative Drug Discovery Group for
Cancer, member, October 25-27, 2004
NIH CSR study section: Basic Mechanisms of Cancer Therapeutics, ad hoc
member, June 2006
NIH CSR study section: Tumor Cell Biology, ad hoc member, June 2007, June 2008
VA Merit Reviewer, Ad Hoc
National Cancer Institute Special Emphasis Panel SPORE in Lymphoma, Brain,
Head/Neck and Lung Cancers, and Sarcoma, February 2012
Alex’s Lemonade Stand Reviewer, 2008 – present
Editorial Boards and Journal Work
Editorial Board Member, Journal of Clinical Oncology and Research, September 2013 present
Editorial Board Member, PeerJ, August 2012 – present
Editorial Board Member, Sarcoma, July 2012 – present
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Founding Editorial Board Member and Associate Editor, Frontiers in Pediatric
Oncology, May 2011 – present
Editorial Board Member, Experimental and Therapeutic Medicine, January 2010 –
present
Editorial Board Member, Pediatric Blood and Cancer, 2007 – present
Manuscript Reviewer, Nature Medicine; Journal of Clinical Investigation, Journal of
Pediatric Hematology and Oncology; Cancer; Cancer Research; Oncogene;
Pediatric Blood and Cancer; Laboratory Investigation; Molecular Cancer
Research; Molecular Cancer Therapeutics, American Journal of Pathology,
Nature, Lancet Oncology
Philanthropy/Organizational
Chairman Medical Advisory Board, Children’s Cancer Foundation, January 2015present
Pediatric Track Chair, American Society of Clinical Oncology Annual Meeting, June
2014
Program Committee, American Society of Clinical Oncology Annual Meeting, June 2012
– 2015
Scientific Advisory Board, Make Some Noise: Cure Kids Cancer Foundation, May
2010 – present
Mentorship Panel, American Society for Clinical Investigation/Association of American
Physicians Joint Meeting, April 2009
Chairman of Medical Board, Dani’s Foundation, 2007– 2014
Children’s Oncology Group, Bone Sarcoma Core Committee, 2005 – 2007
Children’s Oncology Group, Bone Sarcoma Disease Committee, 2001 – present
Children’s Oncology Group, Member of Protocol Committee for AEWS07B1, 2007
American Society of Pediatric Hematology/Oncology, Program Committee, 2003 – 2007
American Association for Cancer Research Annual Meeting Program Committee, 2006
Children’s Oncology Group, Young Investigator Committee, 2001 – 2002
American Society of Pediatric Hematology/Oncology, Membership Committee 1998 –
1999
Medical Advisory Board, Children’s Cancer Foundation, Baltimore, MD, 1997 – 2014
International Service
Reviewer for Israel Cancer Research Fund (ICRF) 2012 – present
Reviewer for Associazione Italiana per la Ricerca sul Cancro (Italien Cancer Society)
2011, 2012, 2014, 2015
Reviewer for Association for International Cancer Research, United Kingdom, 2011
Reviewer for Bone Cancer Research Trust, United Kingdom, 2008, 2009, 2010
Ewing’s Sarcoma International Symposium, April 9-11, 2008, participant
Reviewer for Swiss National Science Foundation, 2006, 2012
Reviewer for Israeli Science Foundation, 2006, 2009, 2010
Ewing’s Sarcoma International Symposium, April 14-16, 2005, co-organizer
Dallas, Texas.
Reviewer for Michael Smith Foundation New Faculty Awards, a British Columbia-based
foundation to support new faculty at BC Universities, 2004.
Ewing’s Sarcoma International Symposium #2, April 22-24, 2002, participant
Virginia.
Ewing’s Sarcoma International Symposium #1, September 25-26, 2000, participant
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Virginia.
Invited Lectures
1. Pediatric Grand Rounds, Memorial Sloan Kettering Cancer Center, 1/30/97, A pathway
of transformation in Ewing’s Family of Tumors via the insulin-like growth factor-I
receptor.
2. Pediatric Oncology Branch Seminar, National Cancer Institute, 2/20/98, Ewing’s
Sarcoma Family of Tumors: Avoiding Apoptosis on the Path Towards Transformation.
3. Maryland Hospital for Children’s Outreach, 11/19/98, Easton Memorial Hospital. ITP:
What Else Can it Be?
4. Easton Memorial Hospital, 11/20/98, Wild Western: Molecular Biology Advances the
Cancer Frontier.
5. Greenebaum Cancer Center Grand Rounds, 3/16/98, Ewing’s Sarcoma Family of
Tumors: Avoiding Apoptosis on the Trail Towards Transformation.
6. Maryland Hospital for Children’s Outreach, 3/17/98, ITP: What Else Can it Be?
7. Mercy Medical Center, 1/8/99, ITP: What Else Can it Be?
8. St. Agnes Hospital Grand Rounds, 2/2000, ITP: What Else Can it Be?
9. Children’s Hospital of Philadelphia, 9/12/00, Ewing’s Sarcoma: What does the
EWS/FLI1 fusion protein do and with whom?
10. Medical College of Virginia, 1/17/01, Wild Western: Molecular Biology Advances the
Cancer Frontier.
11. Hershey Medical Center, 2/25/01, Ewing's Sarcoma: A Model to Unravel the Mysteries
of Oncogenic Transcription.
12. University of Maryland Department of Biochemistry, 9/10/01 EWS/FLI1: A model of
Ewing’s sarcoma.
13. Lombardy Cancer Center, 9/24/01, EWS/FLI1: A Key to Ewing’s Sarcoma.
14. Centre de Recherche de L’Hopital Sainte-Justine, Montreal, Quebec, 11/13/01,
EWS/FLI1: A key to diagnosing and treating Ewing’s Sarcoma.
15. AFLAC Cancer Center Visiting Professor, Emory University, Atlanta, GA, 12/3/01,
EWS/FLI1: A key transcription factor in Ewing’s Sarcoma with therapeutic implications.
16. Georgetown University Department of Pediatrics Grand Rounds, 6/6/03, How are we
hitting the cancer target?
17. Children’s Hospital Los Angeles, Grand Rounds, 4/28/04, Evoking and exploiting
EWS/FLI1 in the therapy of Ewing’s Sarcoma.
18. Pediatric Academic Societies, 5/1/04, Pediatric Malignancies Provide Unique Cancer
Therapy Targets.
19. 18th Annual Pediatric Surgery Symposium at Uniformed Services University of the
Health Sciences, 6/10/04, Molecular Targets in Childhood Cancer: Seek and Destroy.
20. Department of Pediatrics, University of Utah, 3/4/05, Molecular Targets and Ewing’s
Sarcoma.
21. Ewing’s Sarcoma 3rd International Nearburg Symposium, University of Texas
Southwestern Medical Center, Dallas, TX, 4/14 – 4/16/05
22. Pediatric Academic Societies and American Society of Pediatric Hematology/Oncology,
5/14/05, EWS/FLI1: The Perfect Target?
23. Pediatric Grand Rounds, M.D. Anderson Cancer Center, University of Texas, Houston,
TX, 6/20/05, Ewing’s Sarcoma: Can Molecular Therapy Hit a Perfect Target?
24. Cancer Research Institute, University of Texas, San Antonio, TX, 11/1/05, Ewing's
Sarcoma: Can Molecular Therapy Hit a Perfect Target?
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25. Pediatric Grand Rounds, Georgetown University School of Medicine, Washington,
D.C., 5/19/06, New Drugs for Childhood Cancer: How to Throw a Rock Through the
Glass Ceiling.
26. FASEB Phosphatase Meeting, Snowmass, CO, 7/15/06, PTPL1: Is this Phosphatase a
Molecular Target for Ewing’s Sarcoma?
27. Department of Pediatrics, Carver College of Medicine, University of Iowa, Iowa City, IA,
10/24/06, New Drugs for Childhood Cancer: Exploiting Novel Molecular Targets.
28. Child and Family Research Institute, University of British Columbia, Vancouver, BC,
1/11/07, New Drugs for Childhood Cancer: Exploiting Novel Molecular Targets.
29. Sarcoma Mini-Symposium Visiting Lectureship, Huntsman Cancer Institute, University
of Utah, Salt Lake City, UT, 2/14/07, New Drugs for Childhood Cancer: Exploiting
Novel Molecular Targets.
30. National Cancer Institute, Pediatric Oncology Branch Rounds, 2/21/08, New Drugs for
Childhood Cancer: Exploiting Novel Molecular Targets.
31. Broad Institute, MIT, Cambridge, MA, 3/25/08, New Drugs for Childhood Cancer:
Exploiting Novel Molecular Targets.
32. Glaxo-Smith-Kline, Seminar, Collegeville, PA, 3/31/08, New Drugs for Childhood
Cancer: Exploiting Novel Molecular Targets.
33. Rett Nearburg International Ewing’s Sarcoma Symposium IV, Dartmouth College,
Hanover, NH, 4/10/08, EWS-FLI1: Progression from Oncogene to Partner to Poison.
34. PROTHETS, European Ewing’s Sarcoma Project, Rome, Italy, 6/6/08, EWS-FLI1:
Progression from Oncogene to Partner to Poison.
35. Children’s Hospital of Philadelphia, Center for Childhood Cancer Research Seminar
Series, 9/16/08, New Drugs for Childhood Cancer: Exploiting Novel Molecular Targets.
36. Targeted Therapy for Childhood Cancers, Georgetown University Lombardi Cancer
Center, April 15, 2009, Targeted Therapeutics for Ewing’s sarcoma.
37. Distinguished Scientists Seminar Program, Department of Biochemistry and Molecular
Biology, University of South Alabama, Mobile, AL, 4/30/09, EWS-FLI1 to PTPL1: Why
does an oncogene make a phosphatase?
38. Pediatric Cancer Data Portal Workshop, Georgetown University Lombardi Cancer
Center, Washington, DC, 5/18/09. Targeted therapies for pediatric cancers – Ewing’s
Sarcoma.
39. Children's Oncology Group Meeting, Dallas, TX, 9/29/09, Modulation of IGF Signaling
in Ewing’s Sarcoma.
40. University of Minnesota Masonic Cancer Center, Minneapolis, MN, 12/4/09 Small
molecule targeting of the disordered oncoprotein EWS-FLI1 in Ewing's Sarcoma.
41. Children's National Medical Center Research Seminar, Washington, DC, 1/5/10,
Disordered proteins provide amazing small molecule therapeutic targets.
42. Children's National Medical Center Pediatric Grand Rounds, Washington, DC, 1/6/10
Therapeutic targeting of the oncoprotein EWS-FLI1: the Achilles’ Heel of Ewing’s
Sarcoma.
43. Vanderbilt-Ingram Cancer Center, Nashville, TN, 1/7/10, EWS-FLI1 is a therapeutic
target and a disordered protein.
44. Johns Hopkins Translational Medicine Series, Sidney Kimmel Comprehensive Cancer
Center, Baltimore, MD, 1/20/10, Small molecule targeting of EWS-FLI1 to modulate
transcription and create new therapies for Ewing’s Sarcoma.
45. Children’s Hospital of Los Angeles, Los Angeles, CA, 3/10/10, Small molecule
targeting of EWS-FLI1 to modulate transcription and create new therapies for Ewing’s
Sarcoma.
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46. Sarcoma Foundation of America’s Annual Patient Educational Conference, New York,
NY, 4/25/10, Advances in Treatment and Research in Ewing's Sarcoma: Seven Easy
Steps to Make a Targeted Anti-Cancer Drug.
47. American Association for Cancer Research 101st Annual Meeting, Washington, DC,
4/20/10, Therapeutic Targeting of EWS-FLI1: Small Molecule Protein-Protein
Interaction Inhibitors.
48. American Society for Clinical Oncology, Chicago, IL, 6/7/10, Discussion Leader:
Targeting the IGF-IR pathway:Curiosity or Cure?
49. American Society for Clinical Oncology, Chicago, IL, 6/7/10, Educational Lecture: Can
Molecular Targets Translate to Therapeutics? Novel therapeutic opportunities for
sarcomas based upon disordered chromosomal translocation fusion proteins.
50. A.I.Dupont, Wilmington, DE, October 11, 2010, Disorderly Conduct Can Cause Cancer
and Lead to Novel EWS-FLI1 Targeted Therapy.
51. St. Anna’s Kinderspital, Vienna, Austria, November 9, 2010, From Disorderly Conduct
to Small Molecule Targeting EWS-FLI Remains the Villain.
52. Translational Research Interest Group Seminar Series, National Institutes of Health,
Bethesda, MD, February 22, 2011, YK-4-279 is a novel small molecule directly
targeting EWS-FLI1.
53. Georgia Institute of Technology, Atlanta, GA. March 14, 2011, Of biochemists,
synthetic chemists, and molecular biologists: collaboration to discover new drugs for
transcription factor cancer targets.
54. Georgetown University Lombardi Comprehensive Cancer Center, March 30, 2011,
Washington, DC, Drugs and Thugs: Destroying Disordered Proteins.
55. Pharmacology Department, Georgetown University, Washington, DC, May 6, 2011.
56. Memorial Sloan-Kettering Cancer Center, New York, NY, May 19, 2011, Directly
Targeting the Ewing Tumor Stem Cell via EWS-FLI1.
57. Washington Hospital Center, June 10, 2011, Washington, DC, Achieving the Ultimate
Therapeutic Index: Targeting Tumor-Specific Fusion Proteins.
58. Maria Skodowska-Curie Memorial Institute of Oncology, Krakow, Poland, July 4, 2011,
Developing therapies that target oncogenic fusion proteins derived from chromosomal
translocations.
59. Molecular Therapeutics of Cancer Conference, Pacific Grove, CA, July 10-13, 2011,
Drugging the Undruggable, Thinking the Unthinkable.
60. Columbia University Medical Center, New York, NY, July 27, 2011, Small molecules
and intrinsically disordered proteins: Perhaps the next generation of anticancer
therapy.
61. Frontiers in Oncology Lectureship, University of Maryland Greenebaum Cancer Center,
Baltimore, MD, September 21, 2011, Disordered Proteins Cause Challenges, but Open
Opportunities when Targeting Tumorigenic Transcription.
62. 4th Semi-Annual Musculoskeletal Tumor Symposium, Cincinnati, OH, October 17,
2011. New targeted inhibitors for Ewing sarcoma.
63. European Commission-Funded Network on Cancer Research in Children and
Adolescents (ENCCA) Ewing´s Sarcoma Biology Subnetwork meeting Vienna, Austria,
December 20, 2011.
64. Children’s National Medical Center, Washington, DC, January 30, 2012, Drugging the
Undruggable: PK Models and Nuclear Complexes.
65. University of Virginia, Charlottesville, VA. February 2, 2012, A small molecule that
disrupts RNA Helicase A from the oncogenic transcription factor EWS-FLI1 reduces
tumor growth.
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66. Texas Children’s Hospital, Baylor College of Medicine, Houston, TX, March 29, 2012,
Drugging the Undruggable: PK Models Useful to Destroy Nuclear Complexes.
67. AACR Annual Meeting, Chicago, IL, March 31, 2012, Meet-the-Expert Session,
Undruggable Therapeutic Targets and How to Target Them.
68. Transcription and Cancer Meeting, Banbury Center, Cold Spring Harbor Laboratory,
Cold Spring Harbor, NY, April 9-12, 2012, Small-molecule targeting of fusion protein
transcription factors and the biochemistry of intrinsically disordered proteins.
69. Society for Paediatric Oncology and Haematology (Gesellschaft für Pädiatrische
Onkologie und Hämatologie - GPOH) Meeting, Berlin, Germany, May 9-13, 2012,
Keynote Address.
70. National Institutes of Health, Frederick, MD, July 20, 2012, Intrinsically Disordered
Proteins Make Great Drug Targets.
71. Pediatric Grand Rounds, Georgetown University Hospital, Washington, DC, September
7, 2012, Novel treatment for Ewing sarcoma is on the way from idea to clinical trial.
72. European Science Foundation-European Molecular Biology Organization Conference
on Molecular Biology and Innovative Therapies in Sarcomas of Childhood and
Adolescence, Polonia Castle, Pultusk, Poland, September 29 - October 4, 2012,
Targeting fusion protein transcription factors based upon their intrinsic disorder.
73. Cornell University, Ithaca, NY, October 8, 2012, Small-molecule targeting of fusion
protein transcription factors and the biochemistry of intrinsically disordered proteins.
74. Knight Cancer Institute, Oregon Health Sciences University, Portland, OR, October 23,
2012, Drugging the Undruggable: Targeting Transcription in Ewing Sarcoma.
75. Chemotherapy Foundation Symposium XXX, New York, NY, November 6-10, 2012,
Novel approaches to the treatment of Ewing sarcoma.
76. The University of Maryland School of Medicine, Baltimore, MD, January 18, 2013
77. The University of Kansas, Lawrence, KS, January 28, 2013.
78. Cook Children’s Hematology-Oncology Grand Rounds, Forth Worth, TX, February 8,
2013.
79. 8th International Conference on Pediatric Renal Tumor Biology, Bethesda, MD, May
9th, 2013, Keynote Address. Targeting fusion protein transcription factors based upon
their intrinsic disorder.
80. University of Colorado Cancer Center, Denver, CO, May 10th, 2013, Keynote Address.
81. Children’s Oncology Group Sarcoma Biology Session, October 9th, 2013.
82. Rett Nearburg International Ewing’s Sarcoma Symposium V, San Diego, CA,
November 7,2013, EWS-FLI1:Balance and Regulation to Achieve Oncogenesis.
83. Georgetown University Sarah Stewart Lectureship, Feb 6, 2014. Designing a Drug for
the Ideal, but "Undruggable", Cancer Target.
84. Targeted Anti-Cancer Therapy Conference, March 2014. Drugging the Undruggable:
Small Molecule Protein - Protein Interaction Inhibitors
85. Instituto Ortopedico Rizzoli, Bologna, Italy. April 1, 2014. Novel Partner Proteins and
Functions for EWS-FLI1.
86. American Society of Clinical Oncology, Chicago, IL. May, 2014. Pediatric Oncology
Highlights of the Day.
87. American Society of Clinical Oncology, Chicago, IL. May, 2014. Targeted Therapy in
Pediatric Cancer: Where are we now and where do we go from here?
88. Medical College of Virginia/Virginia Commonwealth University, Pediatrics Grand
Rounds, Richmond, VA., May 6, 2014. Targeting EWS-FLI1: The Achilles’ ‘HEAL’ of
Ewing Sarcoma.
89. CureSearch Annual Symposia, Washington, DC. Sept 2014. Targeting the
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‘Undruggable’ EWS-FLI1: Achilles’ ‘HEAL’ of Ewing Sarcoma.
90. Ohio State University, Columbus, OH, Nov 2014. Small Molecule Targeting of EWSFLI1: Killing Cancer While Gaining Mechanistic Insights.
91. Texas Children’s Hospital, Houston, TX April 2015. EWS-FLI1 regulates alternative
splicing: what is the role of splicing in Ewing sarcoma oncogenesis?
92. American Society of Clinical Oncology, Chicago, IL. June, 2015. Poster session
discussant on basic research towards translation.
93. ENCCA Ewing sarcoma meeting, Institute Curie, Paris, France, June 2015.
Challenges to Successfully Targeting EWS-FLI1.
94. St. Jude Children’s Hospital, Memphis, TN, November 2015. Small Molecule Targeting
of EWS-FLI1: Killing Cancer While Gaining Mechanistic Insights.
95. University of Muenster, Departments of Medicine and Pediatric Oncology, Muenster,
Germany, April 2016. Creating a Drug for the Ideal, but "Undruggable", Cancer Target.
96. EuroEwing Strategy Meeting, Muenster, Germany, April 2016. Creating a Drug for the
Ideal, but "Undruggable", Cancer Target.
97. University of Copenhagen, Novo Nordisk Center for Protein Research, Copenhagen,
Denmark, April 2016. Small Molecule Perturbation of EWS-FLI1 Networks: Killing
Cancer While Gaining Mechanistic Insights.
98. Exiqon Life Sciences, Vedbaek, Denmark, April 2016. Small Molecule Perturbation of
EWS-FLI1 Networks: Killing Cancer While Gaining Mechanistic Insights.
99. American Association for Cancer Research, Pediatric Cancer Working Group Special
Scientific Session, New Orleans, LA, April 2016. Creating a Drug for the Ideal, but
"Undruggable", Cancer Target.
100. Gordon Research Conference, Intrisically Disordered Proteins, Les Diablerets,
Switzerland, June 2016.
Georgetown University Service
Program Leader, Molecular Oncology Program (MOP), 2011 to present
Biacore Core Oversight Committee, 2004 to present
Tumor Biology Oversight Committee, 2003 to present
Macromolecular Analysis Core Oversight Committee, 2004 to present
Targeted Therapy for Childhood Cancers, co-chaired/organized symposium that
brought speakers from around country to Georgetown, April 15, 2009
Clinical Faculty Task Force, August 24, 2008
Georgetown University Medical Center Organizational Design Team, 2008 – 2009
Humanomics Symposium Committee Chairman, 2007 – 2008
Georgetown University Medical Center Research Committee, 2005 – 2008
Georgetown University Medical Center Junior Faculty Mentor, 2008
Georgetown University Medical Center Organizational Design Team, 2008
School of Medicine Committee on Medical Education, 2004 – 2005
Drug Discovery Taskforce, 2002 – 2005
Transgenic Animal Imaging Taskforce, 2003 – 2005
Proteomics Task Force, 2004 to 2005
University of Maryland Service
Department of Pediatrics Research Advisory Committee, 1999 – 2002
Program in Molecular and Cellular Biology, Seminar committee, 2001 – 2002
Program in Molecular and Cellular Biology, MD/PhD committee, 2000 – 2002
Program in Oncology, Experimental Therapeutics committee, 1999 – 2002
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Program in Oncology, Drug Discovery Working Group, 2001 – 2002
Dean’s Research Advisory Committee, Chairman Howard Dickler, 2000
Dean’s Task Force on Mentoring, Chairman James Nataro, 1999
Teaching Activities
Georgetown University
Pharmacology Elective in Oncology 2012- present
Small group leader, Medical Student Evidence Based Medicine 2006 to 2012
Course Co-Director, Current Topics in Tumor Biology, 2003 to 2010
Lecturer, Medical Student Year 2 Pathophysiology (hematology and oncology)
Lecturer, Tumor Biology 508, 2003 to present
Small group leader, Medical Student Year 2 Pathophysiology, 2003
Bedside teaching with residents and medical students 4 hours/week and 4 weeks per
year of inpatient attending teaching, 2002 – 2010
Faculty Development Mentor
Aykut Üren, MD, 2002 – present (Dean’s Award for Career Development; Golden
Apple Award for Excellence in Teaching, 2010). Now full Professor.
Joanna Kitlinska, PhD., 2002 – present (Dean’s Award for Career Development),
Promoted to Associate Professor, with Tenure 2015
Brian Rood, MD, NIH K08, 2006 - 2011, Assistant Professor George Washington
University and Attending Children’s National Medical Center
Scott Borinstein, MD., 2009 - 2013., Promoted to Associate Professor.
Rebecca Riggins, PhD., 2013 - present
Chunling Yi, PhD., 2011 – present
Post-Doctoral Fellows
Aykut Uren, MD 1999-2002, tenured Professor Georgetown
Silke Schlottman, PhD, 2006 – 2011; Program Officer at FDA
Nilay Shaw, MD, 2011-2013, Tenure-track position at Ohio State University
Emily Soni, MD, 2010-2013, Tenure Track position at University of Rochester
Hayriye Verda Erkizan, PhD, 2004 – 2015, Research Scientist at VA hospital
Stefan Zoellner, MD, 2013-2016, Faculty University of Muenster, Muenster,
Germany
Yasmine Saygideger, MD., PhD awarded 2015. Faculty, Ismir, Turkey.
Suthee Rapisuwon, MD., 2013- 2015. Research Assistant Professor, GU
Saravana Selvanathan, PhD, 2010 - present
Graduate Students
Julie Barber-Rotenberg, PhD 2012; post-doctoral fellowship Wistar Institute,
Marmorstein Lab
Elspeth Beauchamp, Thesis Committee, PhD 2010; Assistant Professor,
Northwestern University
Ogan Abaan, PhD 2008; post-doctoral fellowship NIH, NCI
Jordan Li, PhD Thesis Committee; PhD 2012
Magdalena Czarnecka, Thesis Committee; PhD 2012
Jean-Baptiste Mazzarti, Thesis Committee; PhD 2012
David Kodack, Thesis Committee, PhD 2009
Jaime Guidry, Thesis Committee Chair, PhD 2009
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Kevin Johnson, Thesis Committee, PhD 2008
Erin Mulbrandt, Thesis Committee, PhD 2008
Tyler Lahusen, Thesis Committee, PhD 2007
Cari Lee, Thesis Committee, PhD 2004, Advisor Todd Waldman
Annabell Oh, Thesis Committee, PhD 2006, Advisor Anton Wellstein
Silvina Frech, Comprehensive Exam 2004
Erin Mulbrandt, Comprehensive Exam 2004
Dora Stylianou, Comprehensive Exam 2003
Margaret Emblom,. Comprehensive Exam 2003
Gregory Maddox, MS 2005
Kelsy McCarty, MS 2004
Dori Paccitil, MS 2003
Rotating Graduate Students
Deanna Tiek, PhD student 2014
Will Kietzman, PhD student 2013
Darius Gaymon, MS student, 2012-2013
Anand Lakhkar, PhD student, 2012
Alana Lelo, MD/PhD student, 2012
Nguyen Nguyen, PhD student, 2011
Tara Gelb, PhD Student, 2011
Rupa Lalchandani, PhD student, 2010
Sonya Parpart, PhD student, 2009
Lymor Ringer, PhD student, 2007
Undergraduate Project Mentees
Sarah Pickard, Georgetown University Howard Hughes Scholar 2004-6
Stanford Medical School 2006 - 2010
Pediatric Residency at Harvard/Boston Children’s Hospital 2010 - 2013
Nilan Schnure, Montgomery Blair Science-Math Magnet Student 2007;
Princeton University Undergraduate 2009 – 2012
University of Pennsylvania Medical School 2013Alison O’Neill, Georgetown University Undergraduate 2008 – 2011
Georgetown Medical School 2012 - present
GU Department of Pediatrics Mentees
Haifa Mtaweh, MD, Completed Pediatric Residency 2010
To begin Pediatric Cardiology at U Toronto/Toronto Sick Children’s
Post-baccalaureate Trainees, POST-laboratory accomplishments:
Sarah Zaidi, Residency in Pediatrics, Texas Children’s Hospital, currently
pediatric pulmonary attending Children’s National Medical Center
Naamah Zitomersky, University of Maryland Medical School 2004, Pediatric
Residency, Fellowship in Pediatric Gastroenterology, Attending
physician Boston Children’s Hospital
Amy Skversky, UMDNJ New Jersey Medical School 2005, Pediatric
Residency, Fellowship Pediatric Nephrology, Ped Nephrologist
Yu-Feng Sun, University of Maryland Medical School 2007, Psychiatry
Residency, practicing psychiatrist
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Amy Levinson, Drexel University Medical School 2008, Pediatric Residency
Fellowship in Pediatric Endocrinology at Boston Children’s Hospital
Kevin Chen, Georgetown University Medical School 2009, Med-Peds Resident
USC
Lauren Scher (Jacobwitz), Georgetown University Medical School 2010,
Anesthesiology residency Johns Hopkins
Alison O’Neill, Georgetown University Medical School 2012
Sarah Ellen Gamble, Georgetown University Medical School 2012
Ryan Commins, Georgetown University Medical School 2012
Faezeh Razjouyan, Howard University Medical School 2012
Garrett Graham, 2012 –
Jeffrey Schneider, 2012 – 2014, MSTP NYU 2014 Summer Intern Mentees
Samantha Halpin, Endocrine Society Fellowship 2003
Brandon Baird, 2003 CURE program
Alex Mazerov, Montgomery Blair Science-Math Magnet Student 2005
Audrey Kubetin, Montgomery Blair Science-Math Magnet Student 2006
Samuel Prager, Montgomery Blair Science-Math Magnet Student 2006
Amber Makani, Georgetown University Howard Hughes Scholar 2007
Charles Kong, Montgomery Blair Science-Math Magnet Student 2008
Eyerusalem Befkadu, University of Maryland-Baltimore County
Undergraduate 2009, PharmD to be completed…
Aaron Plave, Bethesda-Chevy Chase High School Summer 2011
Nadia Turner, Albert Einstein High School Summer 2012, 2013
Sarah Jaffee, University of Rochester, 2013, 2014
Vivian Wang, Poolesville High School Student 2012
Arielle Hollies, Mont County High School, 2013, 2014
Talia Migdal, Washington University 2013, 2014
Alex Hemmer, Albert Einstein High School 2013
Sam Swire, Williams College 2014
Sydney Parks, Thomas Jefferson High School 2015
Jeffrey Blackman, Washinton University 2015
University of Maryland
University of Maryland Pathology 760, 30 students, lecturer 1998 - 2002
Clinic preceptor for Pediatric Hematology/Oncology for year 1 residents, 4
hours/week 1998-2001
Bedside teaching with residents and medical students 4 hours/week, 1997 - present
Medical School Second Year Hematology Lab Instructor, 1998
Graduate Student Committees
Tracy Lessor, PhD awarded 1999
Kara Smolinski, PhD awarded 2000
Dehe Kong, PhD awarded 2000 (thesis reader)
Carrianne Judge , MS awarded 2001
Summer Intern Mentees
Cadence Berrian 2001
Continuing Medical Education Instruction
Toretsky, page
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Pharmaceutical Education and Research Institute (PERI), Arlington, VA
Cancer: Pathophysiology, Current Therapies, Clinical Trials, and Drug
Development, 2003, 2005 - 2010
Cancer 101: A Basic Overview of Current Therapies for New Oncology
Researchers, 2006 – 2009
Scholarship and Research
ACTIVE Research Grants
St. Baldrick’s Foundation (Toretsky - PI)
07/01/14 – 06/30/16 (NCE)
This proposal will establish the full transcriptome of Ewing sarcoma as well as all
expressed alternatively spliced variants. It will directly compare single molecule real
time sequencing (SMRT) using a PacBio instrument to 100 million paired reads from
an Illumina instrument. This proposal will also explore using isoform-specific RNA
hybridization in tumors as a diagnostic and pharmacodynamic biomarker.
Hyuandai Foundation (Toretsky - PI)
07/01/15 - 06/30/17
This proposal supports analyzing the role of YK-4-279 upon metastatic models of
ES alone and in combinatorial therapy.
Allen B. Slifka Foundation (Toretsky - PI)
07/01/15 - 06/30/17
This proposal supports developing pharmacodynamic markers of YK-4-279 and
analogs for clinical development.
5 P30 CA051008-16 (Lou Weiner)
09/30/1990 – 04/30/2019
NIH Cancer Center Support Grant: This grant provides core support for the cancer
center. Toretsky role is Program Leader of Molecular Oncology Program.
COMPLETED RESEARCH SUPPORT
R01 CA138212-01 (Toretsky)
09/01/2008 – 08/31/2015 (NCE)
NIH/NCI
$2,543,385
“Isolation and small molecule targeting of Ewing’s Sarcoma stem cells”
The proposal will identify Ewing’s Sarcoma stem cells and determine the key
signaling pathways used by these cells. In addition, the genetic profiles of the
stem cells will be developed. Small molecules will be used to target the stem cells
to determine if they are differentially sensitive compared to total populations of
tumor cells.
R01CA133662-01A2 (Toretsky)
12/01/2008 – 11/30/2014 (NCE)
NIH/NCI Score 0.8%.
$1,535,000
“Novel Compounds to Inactivate Oncogenic Fusion Proteins”
RNA helicase cooperation with an oncogenic transcription factor is novel, thus we
propose the following hypotheses to interrogate and expand our discovery. We
hypothesize that the interaction of RHA with EWS-FLI1 results in a potent
transcriptional activator/coactivator complex amplifying the functions of both
proteins and together drive the malignant phenotype of ESFT.
1007487 (Toretsky)
Burroughs-Wellcome Foundation
07/01/2008 – 06/30/2015
$750,000
Toretsky, page
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“Novel cancer therapeutics based upon oncogenic fusion-protein transcription
factors”
This project will evaluate small molecules that disrupt RHA from EWS-FLI1 as
potential therapeutics patients with Ewing’s Sarcoma Family of Tumors.
RC4 RFA-OD-10-005
(Toretsky)
09/30/2010 – 08/31/2014
NIH/NCI Score: 1%
$4,373,380
“YK-4-279 specifically targets ETS family fusion-protein cancers in clinical trial”
The overall ambition of this proposal is to optimize delivery methods and obtain
toxicologic data for submission of an investigational new drug (IND) application
leading to a first-in-class, first-in-human clinical trial. The experiments presented in
this proposal will potentially stimulate the opening of a novel area of pharmacologic
development, that of small molecule protein-protein interaction inhibitors (SMPPII)
for transcription.
Creation of a PDGF-C Autocrine Loop by HIC1 Inactivation, Principal
Investigator, Brian R. Rood, Mentor Jeffrey Toretsky, no support, NIH
K08NS051477, 12/01/2006 – 11/30/2011
Signaling Pathways that Determine Ewing’s Sarcoma Outcome, Principal
Investigator Jeffrey Toretsky, NIH/NCI R01 CA88004, 02/01/2000 – 05/31/09.
Total Costs $1,163,632.
Akt Inhibitors to Treat Ewing’s Sarcoma, Principal Investigator Jeffrey
Toretsky, 20% effort. NIH R41 CA102841-01A1, 2 years, total direct costs
$540,592, 08/02/2004 – 08/01/2006.
Shared Instrument Grant for a Biacore T100, Principal Investigator
Jeffrey Toretsky. NIH S10 RR022388-01. Total and direct costs $320,000.
Awarded 02/28/2006.
Targeted regulation of acetylation as novel therapy for Ewing’s sarcoma,
Principal Investigator Jeffrey Toretsky, Alex’s Lemonade Stand Foundation,
07/01/2009 – 06/30/2011, $200,000.
IGFIR in Human GI Tumors, Co-investigator Jeffrey Toretsky, NIH R01
CA78843-01, Total Direct Costs, $834,899. 07/01/1998 - 06/30/2001.
Chemotherapy Resistance in Childhood Malignancy, Principal
Investigator Jeffrey Toretsky, Bear Necessities, $15,000, 7/01/1999 07/01/2000.
Bressler Intramural, Principal Investigator Jeffrey Toretsky, $15,000,
07/01/1999 - 07/01/2000
American Cancer Society Intramural Research Grant, Principal
Investigator Jeffrey Toretsky, $15,000, 07/01/1998 - 07/01/1999
Toretsky, page
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Young Investigator Award, American Society of Clinical Oncology,
Principal Investigator Jeffrey Toretsky, $32,500, 07/01/1998 - 07/01/1999
Outcome Markers for Adolescents with Ewing’s Sarcoma, Principal
Investigator Jeffrey Toretsky. Children’s Oncology Group Adolescent and
Young Adult Committee, 01/01/2007-12/31/2009, direct costs $30,000.
Ewing’s Sarcoma Fusion Protein Interactions, Principal Investigator
Jeffrey Toretsky Children’s Cancer Foundation, 12th year, 12/01/2008 –
11/30/2009, $123,000
Neuropeptide Y in neuroblastoma: growth, angiogenesis and future
therapeutics, PI Joanna Kitlinska, Co-investigator Jeffrey Toretsky, 07/01/2006
– 05/31/2011
No Funding Agency Number
11/01/2010 -10/31/2013
Children’s Cancer Foundation
$75,000
“Revealing the mechanism of YK-4-279’s effect on ESFT cells”
Mentor, Jeffrey Toretsky, no salary support. Grant provides salary to Research
Fellow Hayriye Verda Erkizan, PhD.
Patents-Awarded
Methods and compositions for treating Ewings sarcoma family of tumors
Patent number: 9290449
Abstract: Compounds, compositions and methods relating to EWS-FLI1 protein
inhibitors are provided. The compounds have utility in the treatment of cancers including
the Ewing's sarcoma family of tumors.
Type: Grant
Filed: April 11, 2013
Date of Patent: March 22, 2016
Targeting of EWS-FLI1 as anti-tumor therapy
Patent number: 9045415
Abstract: Peptides and compounds are provided that function as EWS-FLI1 protein
inhibitors. The peptides and compounds have utility in the treatment of Ewing's sarcoma
family of tumors. Also provided are methods of preparing the compounds and assays
for identifying inhibitors of EWS-FLI1 protein.
Type: Grant
Filed: March 9, 2010
Date of Patent: June 2, 2015
Targeting of EWS-FLI1 as anti-tumor therapy
Patent number: 8232310
Abstract: Peptides and compounds are provided that function as EWS-FLI1 protein
inhibitors. The peptides and compounds have utility in the treatment of Ewing's sarcoma
family of tumors. Also provided are methods of preparing the compounds and assays
for identifying inhibitors of EWS-FLI1 protein.
Type: Grant
Filed: June 29, 2009
Toretsky, page
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Date of Patent: July 31, 2012
Patents - Filed/pending
Novel F-18 Labeled Annexin V, Synthesis Thereof, and Use. U.S. Patent Application
60/437,719
Peer-Reviewed Publications
1. Toretsky, JA, Shahidi, NT, Finlay, JL. Effects of Recombinant Human Interferon
Gamma on Hematopoietic Progenitor Cell Growth. Exp. Hematol. 14:182-186 (1986).
2. Rosolen, A., Toretsky, JA, Neckers, LM. Antisense inhibition of CHP-100 c-myc
expression results in reduced in vitro growth kinetics and loss of in vivo tumorigenesis.
Prog. Clin. Biol. Res. 385: 95 - 101 (1994).
3. Rosolen, A., Franscella, E., Toretsky, JA, Neckers, LM. Episome generated c-myc
antisense RNA inhibits growth and tumorigenicity of a human neuroendocrine tumor cell
line. Int. J. of Oncology. 6: 175 - 179 (1995).
4. Toretsky, JA, Neckers, L, Wexler, LH. Detection of the t(11;22)(q24;q12) translocationbearing cells in peripheral blood progenitor cells of patients with Ewing's sarcoma family
of tumors. JNCI 87(5): 385-86 (1995).
5. Blagosklonny MV, Toretsky J, Neckers L. Geldanamycin selectively destabilizes and
conformationally alters mutated p53. Oncogene Sep 7;11(5):933-9 (1995).
6. *Blagosklonny MV, *Toretsky J, Bohen S, Neckers L. Mutant conformation of p53
translated in vitro or in vivo requires functional HSP90. Proceedings of National
Academy of Sciences. 93(16): 8379-83 (1996). (*These authors contributed equally
to this project).
7. Toretsky, JA, Connell, Y, Neckers, L, Bhat, NK. Inhibition of EWS-FLI-1 Fusion Protein
with Antisense Oligodeoxynucleotides. Journal of Neuro-Oncology, 31: 9-16 (1997).
8. Schulte, TW, Toretsky, JA, Ress, E, Helman, L, Neckers, LN. Pax-3 in Ewing’s Family
of Tumors. Biochemical and Molecular Medicine, 60(2): 121-6 (1997).
9. Toretsky, JA, Kalebic, T, Blakesley, V, LeRoith, D and Helman, LJ. The Insulin Like
Growth Factor-I Receptor is Required for EWS/FLI-1 Transformation of Fibroblasts.
Journal of Biological Chemistry, 272(49): 30822-27 (1997).
10. Choo-Kang, LR, Jones, DM, Fehr, JJ, Eskenazi, AE, Toretsky, JA. Cerebral Edema
and priapism in an adolescent with acute lymphblastic leukemia. Pediatric Emergency
Care, 15: 110-2 (1999)
11. Souza, RF, Wang, S, Thakar M, Smolinski, K, Yin, j, Tong-Tong, Z, Kong, D, Abraham,
J, Toretsky, JA, Meltzer, S. Expression of the wild-type insulin-like growth factor-II
receptor gene suppresses growth and causes death in colorectal carcinoma cells.
Oncogene, 18: 4063-68 (1999)
12. Toretsky, JA, Thakar, M, Gutkind, S, Eskenzai, A. Frantz, C. Phosphoinositide 3-OH
kinase blockade enhances apoptosis in Ewing’s Sarcoma Family of Tumors. Cancer
Research, 59: 5745-50 (1999)
13. Young, G, Toretsky, JA, Campbell, A, and Eskenazi, AE. Recognition of Common
Childhood Malignancies. American Journal of Family Practice, 61(7):2144-54. (2000)
14. Toretsky, JA, Zitomersky, N, Eskenazi, A, Voigt, RW, Strauch, ED, Sun, CC, Huber, R,
Meltzer, SJ, and Schlessinger, D. Glypican-3 expression in Wilms tumor and
hepatoblastomas. Journal of Pediatric Hematology and Oncology 23(8): 496-99 (2001).
Toretsky, page
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15. Merino ME, Navid F, Christensen BL, Toretsky JA, Helman LJ, Cheung NV and
Mackall CL. Immunomagnetic purging of Ewing's sarcoma from blood and bone
marrow: quantitation using real-time PCR. Journal of Clinical Oncology, 19:3649-59
(2001)
16. Toretsky, JA, Steinberg, SM, Thakar, M, Zitomersky, N, Counts, DR, Pironis, B,
Parente, C, Eskenazi, AE, Helman, LJ, Wexler, LH. Insulin-Like Growth Factor Type 1
(IGF–1) and IGF Binding Protein–3 in Patients with Ewing Sarcoma Family of Tumors.
Cancer, 92(11):2941-7 (2001).
17. Waldrop C, Kathuria, SS., Toretsky J, and Sun C. Myoepithelioma metastatic to the
orbit. American Journal of Opthalmology, 132(4): 594-6 (2001)
18. Mullins M, Toretsky J, and Rappoport A. PBK/TOPK Is a Novel Mitotic Kinase Which
is Upregulated in Burkitt's Lymphoma and Other Highly Proliferative Malignant Cells.
Blood Cells, Molecules and Diseases 27(5):825-829, (2001).
19. Toretsky, J. A., Jenson, J., Sun, C. C., Eskenazi, A. E., Campbell, A., Hunger, S. P.,
Caires, A., Frantz, C., Hill, J. L., and Stamberg, J. Translocation (11;15;19): a highly
specific chromosome rearrangement associated with poorly differentiated thymic
carcinoma in young patients. Am J Clin Oncol, 26: 300-306, 2003.
20. Toretsky, J. A., Everly, E. M., Padilla-Nash, H. M., Chen, A., Abruzzo, L. V., Eskenazi,
A. E., Frantz, C., Ried, T., and Stamberg, J. Novel Translocation in Acute
Megakaryoblastic Leukemia (AML-M7). J Pediatr Hematol Oncol, 25: 396-402, 2003.
21. Uren, A., Merchant, M. S., Sun, C. J., Vitolo, M. I., Sun, Y., Tsokos, M., Illei, P. B.,
Ladanyi, M., Passaniti, A., Mackall, C., and Toretsky, J. A. Beta-platelet-derived growth
factor receptor mediates motility and growth of Ewing's sarcoma cells. Oncogene, 22:
2334-2342, 2003.
22. Gorlick, R., Anderson, P., Andrulis, I., Arndt, C., Beardsley, G. P., Bernstein, M., Bridge,
J., Cheung, N. K., Dome, J. S., Ebb, D., Gardner, T., Gebhardt, M., Grier, H., Hansen,
M., Healey, J., Helman, L., Hock, J., Houghton, J., Houghton, P., Huvos, A., Khanna,
C., Kieran, M., Kleinerman, E., Ladanyi, M., Lau, C., Malkin, D., Marina, N., Meltzer, P.,
Meyers, P., Schofield, D., Schwartz, C., Smith, M. A., Toretsky, J., Tsokos, M., Wexler,
L., Wigginton, J., Withrow, S., Schoenfeldt, M., and Anderson, B. Biology of Childhood
Osteogenic Sarcoma and Potential Targets for Therapeutic Development: Meeting
Summary. Clin Cancer Res, 9: 5442-5453, 2003.
23. Gober, M. D., Smith, C. C., Ueda, K., Toretsky, J. A., and Aurelian, L. Forced
expression of the H11 heat shock protein can be regulated by DNA methylation and
trigger apoptosis in human cells. J Biol Chem, 278: 37600-37609, 2003.
24. Zhang, H., Merchant, M. S., Chua, K. S., Khanna, C., Helman, L. J., Telford, B., Ward,
Y., Summers, J., Toretsky, J., Thomas, E. K., June, C. H., and Mackall, C. L. Tumor
Expression of 4-1BB Ligand Sustains Tumor Lytic T Cells. Cancer Biol Ther, 2: 579586, 2003.
25. Jeffrey A. Toretsky, Amy Levenson, Irvin N. Weinberg, Jonathan F. Tait, Aykut Üren,
and Ronald C. Mease. Preparation of F-18 labeled annexin V: a potential PET
radiopharmaceutical for imaging cell death. Nuclear Medicine and Biology, 31(6):74752, 2004.
26. Aykut Üren, Vladimir Wolf, Yu-Feng Sun, Amir Azari, Jeffrey S. Rubin, Jeffrey A
Toretsky. Wnt/Frizzled Signaling in Ewing’s Sarcoma. Pediatric Blood and Cancer,
43(3): 243-9, 2004.
27. Uren, A., Tcherkasskaya, O., and Toretsky, J. A. Recombinant EWS-FLI1 oncoprotein
activates transcription. Biochemistry, 43: 13579-13589, 2004.
Toretsky, page
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28. French, C. A., Kutok, J. L., Faquin, W. C., Toretsky, J. A., Antonescu, C. R., Griffin, C.
A., Nose, V., Vargas, S. O., Moschovi, M., Tzortzatou-Stathopoulou, F., Miyoshi, I.,
Perez-Atayde, A. R., Aster, J. C., and Fletcher, J. A. Midline carcinoma of children and
young adults with NUT rearrangement. J Clin Oncol, 22: 4135-4139, 2004.
29. Kitlinska, J., Abe, K., Kuo, L., Pons, J., Yu, M., Li, L., Tilan, J., Everhart, L., Lee, E. W.,
Zukowska, Z., and Toretsky, J. A. Differential effects of neuropeptide Y on the growth
and vascularization of neural crest-derived tumors. Cancer Res, 65: 1719-1728, 2005.
30. Abaan, O. D., Levenson, A., Khan, O., Furth, P. A., Üren, A., and Toretsky, J. A.
PTPL1 is a Direct Transcriptional Target of EWS/FLI1 and Modulates Ewing’s Sarcoma
Tumorigenesis. Oncogene, 24: 2715-2722, 2005.
31. Üren, A., Fallen, S., Yuan, H., Usubutun, A., Kucukali, T., Schlegel, R., and Toretsky,
J. A.Activation of the Canonical Wnt Pathway during Genital Keratinocyte
Transformation: A Model for Cervical Cancer Progression. Cancer Res, 65: 6199-6206,
2005.
32. Brooks, B. P., Meck, J. M., Haddad, B. R., Bendavid, C., Blain, D., and Toretsky, J. A.
Factor VII deficiency and developmental abnormalities in a patient with partial
monosomy of 13q and trisomy of 16p: case report and review of the literature. BMC
Med Genet, 7: 2, 2006. PMCID: PMC1379634
33. Toretsky, J. A., Erkizan, V., Levenson, A., Abaan, O. D., Parvin, J. D., Cripe, T. P.,
Rice, A. M., Lee, S. B., and Uren, A. Oncoprotein EWS-FLI1 activity is enhanced by
RNA helicase A. Cancer Res, 66: 5574-5581, 2006.
34. Kitlinska, J., Kuo, L., Abe, K., Pons, J., Yu, M., Li, L., Tilan, J., Toretsky, J., and
Zukowska, Z. Role of neuropeptide Y and dipeptidyl peptidase IV in regulation of
Ewing's sarcoma growth. Adv Exp Med Biol, 575: 223-229, 2006.
35. Adhikari, S., Toretsky, J. A., Yuan, L., and Roy, R. Magnesium, essential for base
excision repair enzymes, inhibits substrate binding of N-methylpurine-DNA glycosylase.
J Biol Chem, 281: 29525-32, 2006.
36. Aryee, D. N., Kreppel, M., Bachmaier, R., Uren, A., Muehlbacher, K., Wagner, S.,
Breiteneder, H., Ban, J., Toretsky, J. A., and Kovar, H. Single-chain Antibodies to the
EWS NH2 Terminus Structurally Discriminate between Intact and Chimeric EWS in
Ewing's Sarcoma and Interfere with the Transcriptional Activity of EWS In vivo. Cancer
Res, 66: 9862-9869, 2006.
37. French CA, Ramirez CL, Kolmakova J, Hickman TT, Cameron MJ, Thyne ME, Kutok JL,
Toretsky JA, Tadavarthy AK, Kees UR, Fletcher JA, Aster JC. BRD-NUT oncoproteins:
a family of closely related nuclear proteins that block epithelial differentiation and
maintain the growth of carcinoma cells. Oncogene, 27(15):2237-42, 2008.
38. Endo Y, Beauchamp E, Woods D, Taylor WG, Toretsky JA, Uren A, Rubin JS. Wnt-3a
and Dickkopf-1 stimulate neurite outgrowth in Ewing tumor cells via a Frizzled3- and cJun N-terminal kinase-dependent mechanism. Mol Cell Biol, 28(7):2368-79, 2008.
PMCID: PMC2268413
39. Chen K, Fallen S, Abaan HO, Hayran M, Gonzalez C, Wodajo F, MacDonald T,
Toretsky JA, Uren A. Wnt10b induces chemotaxis of osteosarcoma and correlates with
reduced survival. Pediatr Blood Cancer, 51(3):349-55, 2008.
40. Beauchamp E, Bulut G, Abaan OD, Chen K, Merchant A, Matsui W, Endo Y, Rubin JS,
Toretsky JA, Uren A. GLI1 is a direct transcriptional target of EWS-FLI1 oncoprotein. J
Biol Chem 284(14):9074-82, 2009. PMCID: PMC2666556
41. Stylianou DC, Auf der Maur A, Kodack DP, Henke RT, Hohn S, Toretsky JA, Riegel
AT, Wellstein A. Effect of single-chain antibody targeting of the ligand-binding domain in
the anaplastic lymphoma kinase receptor. Oncogene, 28(37):3296-306, 2009.
Toretsky, page
18
42. Raygada M, Arthur DC, Wayne AS, Rennert OM, Toretsky JA, Stratakis CA. Juvenile
xanthogranuloma in a child with previously unsuspected neurofibromatosis type 1 and
juvenile myelomonocytic leukemia. Pediatr Blood Cancer, 2009. PMCID: PMC2783853
43. Erkizan HV, Kong Y, Merchant M, Schlottmann S, Barber-Rotenberg JS, Yuan L, Abaan
OD, Chou TH, Dakshanamurthy S, Brown ML, Uren A, Toretsky JA. A small molecule
blocking oncogenic protein EWS-FLI1 interaction with RNA helicase A inhibits growth of
Ewing's sarcoma. Nature Medicine, 15(7):750-6, 2009. PMCID: PMC2777681.
EDITOR’S CHOICE REVIEW IN SCIENCE, AUGUST 2009. FACULTY OF 1000
EXCEPTIONAL ARTICLE LIST WITH AN F1000 FACTOR SCORE OF 9.0.
44. Lu C, Everhart L, Tilan J, Kuo L, Sun CC, Munivenkatappa, R. B., Jonsson-Rylander, A.
C., Sun, J., Kuan-Celarier, A., Li, L., Abe, K., Zukowska, Z., Toretsky, J. A., Kitlinska, J.
Neuropeptide Y and its Y2 receptor: potential targets in neuroblastoma therapy.
Oncogene 29: 5630-5642, 2010. PMCID: PMC2955165
45. Awad O, Yustein JT, Shah P, Gul N, Katuri V, Alison O’Neill, Yali Kong, Milton L. Brown,
*Jeffrey A. Toretsky, *David M. Loeb. High ALDH Activity Identifies ChemotherapyResistant Ewing’s Sarcoma Stem Cells That Retain Sensitivity to EWS-FLI1 Inhibition.
PLoS ONE 5(11): e13943. 2010 * Co-senior authored. PMCID: PMC2978678
46. Beauchamp EM, Ringer L, Bulut G, Sajwan KP, Hall MD, Lee, Y. C., Peaceman, D.,
Ozdemirli, M., Rodriguez, O., Macdonald, T. J., Albanese, C., Toretsky, J. A., Uren, A.
Arsenic trioxide inhibits human cancer cell growth and tumor development in mice by
blocking Hedgehog/GLI pathway. J Clin Invest 121(1): 148-160, 2011.
PMCID: PMC3007144
47. Borinstein SC, Barkauskas DA, Krailo M, Scher D, Scher L, Schlottmann S, Kallakury B,
Dickman PS, Pawel BR, West DC, Womer RB, Toretsky JA. Investigation of the
Insulin-Like Growth Factor-1 signaling pathway in localized Ewing sarcoma: a report
from the Children’s Oncology Group. Cancer 117(21):4966-76, 2011.
48. Rahim S, Beauchamp EM, Kong Y, Brown ML, Toretsky JA, Üren A. YK-4-279 inhibits
ERG and ETV1 mediated prostate cancer cell invasion. PLoS One. 2011 Apr
29;6(4):e19343. PMCID: PMC3084826
49. Lu C, Tilan JU, Everhart L, Czarnecka M, Soldin SJ, Mendu DR, Jeha D, Hanafy J, Lee
CK, Sun J, Izycka-Swieszczewska E, Toretsky JA, Kitlinska J. Dipeptidyl peptidases
as survival factors in Ewing's sarcoma family of tumors: implications for tumor biology
and therapy. J Biol Chem. 2011 Aug 5;286(31):27494-505.. PMCID: PMC3149342
50. Bulut G, Hong SH, Chen K, Beauchamp EM, Rahim S, Kosturko GW, Glasgow E,
Dakshanamurthy S, Lee HS, Daar I, Toretsky JA, Khanna C, Uren A. Small molecule
inhibitors of ezrin inhibit the invasive phenotype of osteosarcoma cells. Oncogene
31(3):269-81, 2012. PMCID: PMC3513970
51. Solomon DA, Kim T, Diaz-Martinez LA, Fair J, Elkahloun AG, Harris BT, Toretsky J,
Rosenberg SA, Shukla N, Ladanyi M, Samuels Y, James CD, Yu H, Kim JS, Waldman
T. Mutational inactivation of STAG2 causes aneuploidy in human cancer. Science
333(6045):1039-43, 2011. PMCID: PMC3374335
52. Erkizan HV, Scher LJ, Gamble E, Barber-Rotenberg JS, Sajwan KP, Üren A, Toretsky
JA. Novel peptide binds EWS-FLI1 and reduces the oncogenic potential in Ewing
tumors. Cell Cycle 10(19):3397-408, 2011. PMCID: PMC3233630
53. Gülay Bulut, Shannon Fallen, Elspeth Beauchamp, Lauren Elizabeth Drebing, Junfeng
Sun, Deborah L. Berry, Bhaskar Kallakury, Christopher P. Crum, Jeffrey A. Toretsky,
Richard Schlegel, Aykut Üren. Beta-Catenin Accelerates Human Papilloma Virus Type16 Mediated Cervical Carcinogenesis in Transgenic Mice. PLoS ONE 6(11): e27243,
2011. PMCID: PMC3210148
Toretsky, page
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54. Ringer L, Sirajuddin P, Heckler M, Ghosh A, Suprynowicz F, Yenugonda VM, Brown
ML, Toretsky JA, Uren A, Lee Y, Macdonald TJ, Rodriguez O, Glazer RI, Schlegel R,
Albanese. VMY-1-103 is a novel CDK inhibitor that disrupts chromosome organization
and delays metaphase progression in medulloblastoma cells. Cancer Biol Ther.
12(9):818-26, 2011. PMCID: PMC3367670
55. Scott Grindrod, Simeng Suy, Shannon Fallen, Masumi Eto, Jeffery Toretsky, Milton L.
Brown. Effects of a Fluorescent Myosin Light Chain Phosphatase Inhibitor on Prostate
Cancer Cells. Front Oncol 1:27, 2011. PMCID: PMC3356144
56. Smith MA, Kang MH, Reynolds CP, Kurmasheva RT, Alexander D, Billups CA,
Toretsky JA, Houghton PJ. Evaluation of arsenic trioxide by the pediatric preclinical
testing program with a focus on Ewing sarcoma. Pediatr Blood Cancer 59(4): 753–755,
2012. PMCID: PMC3612422
57. Barber-Rotenberg JS, Selvanathan SP, Kong Y, Erkizan HV, Snyder TM, Hong SP,
Kobs CL, South NL, Summer S, Monroe PJ, Chruszcz M, Dobrev V, Tosso PN, Scher
LJ, Minor W, Brown ML, Metallo SJ, Üren A, Toretsky JA. Single Enantiomer of YK-4279 Demonstrates Specificity in Targeting the Oncogene EWS-FLI1. Oncotarget
3(2):172-82, 2012. PMCID: PMC3326647
58. Heinrich Kovar, Javier Alonso, Pierre Aman, Dave N.T. Aryee, Jozef Ban, Sue Burchill,
Stefan Burdach, Enrique De Alava, Olivier Delattre, Uta Dirksen, Argyro Fourtouna,
Simone Fulda, Lee J. Helman, David Herrero-Martin, Pancras C.W. Hogendoorn, Udo
Kontny, Elizabeth R. Lawlor, Stephen L. Lessnick, Antonio Llombart-Bosch, Markus
Metzler, Richard Moriggl, Stephan Niedan, Jenny Potratz, Françoise Redini, Günther
H.S. Richter, Lucia T. Riedmann, Claudia Rossig, Beat W. Schäfer, Raphaela
Schwentner, Katia Scotlandi, Poul H. Sorensen, Martin S. Staege, Franck Tirode,
Jeffrey Toretsky, Selena Ventura, Angelika Eggert, Ruth Ladenstein. The First
European Interdisciplinary Ewing sarcoma research summit. Frontiers in Pediatric
Oncology. 2:54, 2012. Epub 2012 May 29. PMCID: PMC3361960
59. Schlottmann S, Erkizan HV, Barber-Rotenberg JS, Knights C, Cheema A, Üren A,.
Avantaggiati ML, Toretsky, JA. Acetylation increases EWS-FLI1 DNA binding and
transcriptional activity. Frontiers in Pediatric Oncology 2:107, 2012.
PMCID: PMC3435532
60. Ogan D Abaan, Wiljan Hendriks, Aykut Üren, Jeffrey A. Toretsky, Hayriye V Erkizan.
Valosin containing protein (VCP/p97) is a novel substrate for the protein tyrosine
phosphatase PTPL1. Experimental Cell Research 319(1): 1-11, 2013.
PMCID: PMC3638858
61. Kirilyuk A, Shimoji M, Catania J, Sahu G, Pattabiraman N, Giordano A, Albanese C,
Mocchetti I, Toretsky JA, Uversky VN, Avantaggiati ML. An Intrinsically Disordered
Region of the Acetyltransferase p300 with Similarity to Prion-Like Domains Plays a Role
in Aggregation. PLoS One 7(11):e48243, 2012. PMCID: PMC3486812
62. Alison O'Neill, Nilay Shah, Naamah Zitomersky, Marc Ladanyi, Neerav Shukla, Aykut
Üren, David Loeb, and Jeffrey Toretsky. Insulin-Like Growth Factor 1 Receptor as a
Therapeutic Target in Ewing Sarcoma: Lack of Consistent Upregulation or Recurrent
Mutation and a Review of the Clinical Trial Literature. Sarcoma. 2013: 450478
PMCID: PMC3569907
63. Lee HJ, Yoon C, Schmidt B, Park do J, Zhang AY, Erkizan HV, Toretsky JA, Kirsch
DG, Yoon SS. Combining PARP-1 inhibition and radiation in ewing sarcoma results in
lethal DNA damage. Mol Cancer Ther. 2013 Nov;12(11):2591-600.
Toretsky, page
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64. Paige M, Kosturko G, Bulut G, Miessau M, Rahim S, Toretsky JA, Brown ML, Uren A.
Design, synthesis and biological evaluation of ezrin inhibitors targeting metastatic
osteosarcoma. Bioorg Med Chem. 2014 Jan 1;22(1):478-87.
65. Tilan JU, Lu C, Galli S, Izycka-Swieszewska E, Earnest JP, Shabbir A, Everhart LM,
Wang S, Martin S, Horton M, Mahajan A, Christian D, O'Neill A, Wang H, Zhuang T,
Czarnecka M, Johnson MD, Toretsky JA, Kitlinska J. Hypoxia shifts activity of
neuropeptide Y in Ewing sarcoma from growth-inhibitory to growth-promoting effects.
Oncotarget. 2013 Dec;4(12):2487-501.
66. Sung-Hyeok Hong, Sarah. E. Youbi, S. Peter Hong, Bhaskar Kallakury, Phillip Monroe,
Hayriye V Erkizan, Julie S. Barber-Rotenberg, Peter Houghton, Aykut Üren, Jeffrey A.
Toretsky. Pharmacokinetic modeling optimizes inhibition of the ‘undruggable’ EWSFLI1 transcription factor in Ewing Sarcoma. Oncotarget. 2014 Jan; 5(2):338-50.
67. Borinstein SC, Barkauskas DA, Bernstein M, Goorin A, Gorlick R, Krailo M, Schwartz
CL, Wexler LH, Toretsky JA. Analysis of serum insulin growth factor-1 concentrations
in localized osteosarcoma: a children's oncology group study. Pediatr Blood Cancer.
2014 Apr;61(4):749-52.
68. Carmody Soni EE, Schlottman S, Erkizan HV, Uren A, Toretsky JA. Loss of SS18SSX1 inhibits viability and induces apoptosis in synovial sarcoma. Clin Orthop Relat
Res. 2014 Mar;472(3):874-82.
69. Zhang W, Nandakumar N, Shi Y, Manzano M, Smith A, Graham G, Gupta S, Vietsch
EE, Laughlin SZ, Wadhwa M, Chetram M, Joshi M, Wang F, Kallakury B, Toretsky J,
Wellstein A, Yi C. Downstream of mutant KRAS, the transcription regulator YAP is
essential for neoplastic progression to pancreatic ductal adenocarcinoma. Science
Signaling. 2014 May 6;7(324):ra42. PMC:4175524
70. Rahim S, Minas T, Hong SH, Justvig S, Celik H, Kont YS, Han J, Kallarakal AT, Kong Y,
Rudek MA, Brown ML, Kallakury B, Toretsky JA, Uren A. A Small Molecule Inhibitor of
ETV1, YK-4-279, Prevents Prostate Cancer Growth and Metastasis in a Mouse
Xenograft Model. PLoS ONE. 2014;9(12):e114260. PMC:4257561
71. Shah N, Wang J, Selich-Anderson J, Graham G, Siddiqui H, Li X, Khan J, Toretsky J.
PBX1 Is a Favorable Prognostic Biomarker as It Modulates 13-cis Retinoic AcidMediated Differentiation in Neuroblastoma. Clin Cancer Res. 2014 Aug 15;20(16):440012. PMC:4134768
72. Tosso PN, Kong Y, Scher L, Cummins R, Schneider J, Rahim S, Holman KT, Toretsky
J, Wang K, Uren A, Brown ML. Synthesis and Structure-Activity Relationship Studies of
Small Molecule Disruptors of EWS-FLI1 Interactions in Ewing's Sarcoma. J Med Chem.
2014 Dec 26;57(24):10290-303. PMC:4281097
73. Tilan JU, Krailo M, Barkauskas DA, Galli S, Mtaweh H, Long J, Wang H, Hawkins K, Lu
C, Jeha D, Izycka-Swieszewska E, Lawlor ER, Toretsky JA, Kitlinska JB. Systemic
levels of neuropeptide Y and dipeptidyl peptidase activity in patients with Ewing
sarcoma--associations with tumor phenotype and survival. Cancer. 2015 Mar
1;121(5):697-707. PubMed PMID: 25387699. Pubmed Central PMCID: 4339539.
74. Erkizan HV, Schneider JA, Sajwan K, Graham GT, Griffin B, Chasovskikh S, Youbi SE,
Kallarakal A, Chruszcz M, Padmanabhan R, Casey JL, Uren A, Toretsky JA. RNA
helicase A activity is inhibited by oncogenic transcription factor EWS-FLI1. Nucleic
Acids Res. 2015 Jan;43(2):1069-80. PubMed PMID: 25564528. Pubmed Central
PMCID: 4333382.
75. Hong SH, Tilan JU, Galli S, Izycka-Swieszewska E, Polk T, Horton M, Mahajan A,
Christian D, Jenkins S, Acree R, Connors K, Ledo P, Lu C, Lee YC, Rodriguez O,
Toretsky JA, Albanese C, Kitlinska J. High neuropeptide Y release associates with
Toretsky, page
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Ewing sarcoma bone dissemination - in vivo model of site-specific metastases.
Oncotarget. 2015 Mar 30;6(9):7151-65. PubMed PMID: 25714031. Pubmed Central
PMCID: 4466675.
76. Celik H, Hong SH, Colon-Lopez DD, Han J, Kont YS, Minas TZ, Swift M, Paige M,
Glasgow E, Toretsky JA, Bosch J, Uren A. Identification of Novel Ezrin Inhibitors
Targeting Metastatic Osteosarcoma by Screening Open Access Malaria Box. Mol
Cancer Ther. 2015 Nov;14(11):2497-507. PubMed PMID: 26358752. Pubmed Central
PMCID: 4636458.
77. Stieglitz E, Taylor-Weiner AN, Chang TY, Gelston LC, Wang YD, Mazor T, Esquivel E,
Yu A, Seepo S, Olsen SR, Rosenberg M, Archambeault SL, Abusin G, Beckman K,
Brown PA, Briones M, Carcamo B, Cooper T, Dahl GV, Emanuel PD, Fluchel MN,
Goyal RK, Hayashi RJ, Hitzler J, Hugge C, Liu YL, Messinger YH, Mahoney DH, Jr.,
Monteleone P, Nemecek ER, Roehrs PA, Schore RJ, Stine KC, Takemoto CM,
Toretsky JA, Costello JF, Olshen AB, Stewart C, Li Y, Ma J, Gerbing RB, Alonzo TA,
Getz G, Gruber TA, Golub TR, Stegmaier K, Loh ML. The genomic landscape of
juvenile myelomonocytic leukemia. Nat Genet. 2015 Nov;47(11):1326-33. PubMed
PMID: 26457647. Pubmed Central PMCID: 4626387.
78. Minas TZ, Han J, Javaheri T, Hong SH, Schlederer M, Saygideger-Kont Y, Celik H,
Mueller KM, Temel I, Ozdemirli M, Kovar H, Erkizan HV, Toretsky J, Kenner L, Moriggl
R, Uren A. YK-4-279 effectively antagonizes EWS-FLI1 induced leukemia in a
transgenic mouse model. Oncotarget. 2015 Nov 10;6(35):37678-94. PubMed PMID:
26462019. Pubmed Central PMCID: 4741957.
79. Kovar H, Amatruda J, Brunet E, Burdach S, Cidre-Aranaz F, de Alava E, Dirksen U, van
der Ent W, Grohar P, Grunewald TG, Helman L, Houghton P, Iljin K, Korsching E,
Ladanyi M, Lawlor E, Lessnick S, Ludwig J, Meltzer P, Metzler M, Mora J, Moriggl R,
Nakamura T, Papamarkou T, Radic Sarikas B, Redini F, Richter GH, Rossig C,
Schadler K, Schafer BW, Scotlandi K, Sheffield NC, Shelat A, Snaar-Jagalska E,
Sorensen P, Stegmaier K, Stewart E, Sweet-Cordero A, Szuhai K, Tirado OM, Tirode F,
Toretsky J, Tsafou K, Uren A, Zinovyev A, Delattre O. The second European
interdisciplinary Ewing sarcoma research summit - A joint effort to deconstructing the
multiple layers of a complex disease. Oncotarget. 2016 Feb 23;7(8):8613-24. PubMed
PMID: 26802024.
80. Saygideger-Kont Y, Minas TZ, Jones H, Hour S, Celik H, Temel I, Han J, Atabey N,
Erkizan HV, Toretsky JA, Uren A. Ezrin Enhances EGFR Signaling and Modulates
Erlotinib Sensitivity in Non-Small Cell Lung Cancer Cells. Neoplasia. 2016
Feb;18(2):111-20. PubMed PMID: 26936397.
Peer-Reviewed Reviews
1. Toretsky, JA and Helman LJ. Insulin-like Growth Factor II and Human Cancer. Journal
of Endocrinology 149(3): 367-372, 1996.
2. Toretsky, JA and Helman, LJ. Cytogenetics and Experimental Models in Current
Opinion in Oncology, Sarcomas, ed Holcombe Grier, Rapid Science Press, 342-7,
1997.
3. Toretsky, JA. Insulin-like Growth Factor Type-I Receptor Signaling. Cancer Research
Alert, Cover Article, June 2000.
4. Üren, A. and Toretsky, J. A. Pediatric malignancies provide unique cancer therapy
targets. Curr Opin Pediatr, 17: 14-19, 2005.
5. Üren, Aykut and Toretsky, Jeffrey A. Ewing’s sarcoma oncoprotein EWS-FLI1: the
perfect target without a therapeutic agent. Future Oncology, 1(4):521-528, 2005.
Toretsky, page
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6.
Abaan OD, Toretsky JA. PTPL1: a large phosphatase with a split personality. Cancer
Metastasis Rev, 27(2):205-14, 2008.
7. Kim SY, Toretsky JA, Scher D, Helman LJ. The Role of IGF-1R in Pediatric
Malignancies. Oncologist, 2009. PMCID: PMC2721475
8. Hayriye V. Erkizan, Vladimir N. Uversky, Jeffrey A. Toretsky, Oncogenic Partnerships:
EWS-FLI1 Protein Interactions Initiate Key Pathways of Ewing’s Sarcoma. Clin Cancer
Res 16, 4077-4083, 2010.
9. Toretsky JA, Wright PE. Assemblages: Functional units formed by cellular phase
separation. J Cell Biol. 2014 Sep 1;206(5):579-88.
10. Zollner SK, Rossig C, Toretsky JA. Synovial sarcoma is a gateway to the role of
chromatin remodeling in cancer. Cancer Metastasis Rev. 2015 Sep;34(3):417-28.
PubMed PMID: 26277104.
Invited Commentary
Toretsky, Jeffrey A and Gorlick, Richard. IGF-IR targeted treatment of sarcoma. Lancet
Oncology, 11(2): 105-106, 2010.
Wellstein A, Toretsky JA. Hunting ALK to feed targeted cancer therapy. Nat Med. 2011
Mar;17(3):290-1.
Book and Online Chapters
Gorlick, R, Toretsky, J, Teot, L, Bernstein, M. “Bone Tumors”, in Holland and Frei Cancer
Medicine, 7th edition, Editors Kufe, Pollack, Weichselbaum, Bast, Gansler, Holland, and Frei,
April 2006.
Gorlick, R, Toretsky, J, Teot, L, Bernstein, M. “Bone Tumors,” in Holland and Frei Cancer
Medicine, 6th edition, Editors Kufe, Pollack, Weichselbaum, Bast, Gansler, Holland, and Frei,
April 2003.
Toretsky, J. “Ewing Sarcoma and Primitive Neuroectodermal Tumors.” Epediatrics,
Emedicine, 2001, Updates 2005, 2007, 2008, 2012, 2014. http://www.epediatrics.com.
11,543 page views as of 1/26/10.
Jeffrey A. Toretsky, Heinrich Kovar, Michael Paulussen, R. Lor Randall, Andreas Schuck,
Lisa A. Teot, Herbert Jurgens and Mark Bernstein. Ewing’s Sarcoma Family of Tumors, in
Cancer in Children, 1st edition, Editor William Carroll, Jones and Bartlett Publishers, 2007.
Toretsky, Jeffrey A. and Uren, Aykut. “Ewing’s Sarcoma Family of Tumors: Molecular Targets
Need Arrows,” in Molecularly Targeted Therapy for Childhood Cancer, 1st Edition, Editors,
Peter Houghton and Robert Arceci, Springer Science and Business Media, 2010.
Toretsky, Jeffrey A and Mackall, Crystal. “Ewing’s Sarcoma Family of Tumors”, in Rudolph’s
Pediatrics, Editors, Colin D. Rudolph, et al., McGraw Hill Medical, 2010.
Toretsky, Jeffrey A., Heinrich Kovar, Michael Paulussen, R. Lor Randall, Andreas Schuck,
Lisa A. Teot, Herbert Jurgens and Mark Bernstein. “Ewing’s Sarcoma Family of Turmors.”
Cancer in Children and Adolescents. Ed. William L Carroll and Jonathan L Finlay. Sudbury,
Mass.: Jones and Bartlett Publishers, 2010. 395-408.
Toretsky, page
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Jeffrey A. Toretsky and Aerang Kim. “ Pediatric Solid Tumors: Embryonal Cell Oncogenesis”.
in Molecular Oncology: Causes of Cancer and Targets for Treatment., edited by Edward
Gelmann, Charles Sawyers, and Frank Rauscher III. Cambridge University Press, 826-35,
2014.
Meeting Abstracts
1. Toretsky, JA, Shahidi, NT, Finlay, JL. Effects of Human Interferon Gamma on Human
Hematopoietic Progenitor Cells. International Society of Experimental Hematology,
August 1984.
2. Finlay, JL, Toretsky, JA, Hoffman, R, Bruno, E, Shahidi, NT. Cyclosporine A in
Refractory Aplastic Anemia. American Society of Hematology, December 1984.
3. Toretsky, JA, Whitesell, L, Neckers, LM. Use of Geldanamycin to Study Early Activation
Events in Normal Lymphocytes. Molecular Basis of Cancer. June 1993.
4. Toretsky, JA, Bhat, NK, Neckers, LM. Inhibition of EWS-ERG-B Protein Alters Tumor
Growth of Peripheral Primitive Neuroectodermal Tumors in Athymic Mice. Molecular
Basis of Cancer, June 1994.
5. Toretsky, JA, Neckers, L, Wexler, LH. Minimal Residual Disease in the Ewing's
Sarcoma Family of Tumors: Evaluation of Peripheral Blood Progenitor Cells. Abstract.
American Society of Pediatric Hematology/Oncology. October 1994.
6. Toretsky, JA, Connell, Y, Neckers, L, Bhat, NK. Inhibition of EWS-FLI-1 Fusion Protein
with Antisense Oligodeoxynucleotides. Biochemistry of Neuroectodermal Tumors, Rome
Itlay, June 1995.
7. Toretsky, JA, Bachman, C, Kalebic, T, Blakesley, V, LeRoith, D, Helman, L. Insulin-like
Growth Factor-I Receptor is Necessary for EWS/FLI-1 Induced Transformation of
Fibroblasts. Transcription Factors in Cancer. American Association of Cancer Research,
January 1997.
8. Blakesley, V, Helman, L, Kalebic, T, LeRoith, D, Toretsky, JA. Insulin-like Growth
Factor-I Receptor is Necessary for EWS/FLI-1 Induced Transformation of Fibroblasts.
American Society of Pediatric Hematology/Oncology, Chicago, IL, October 1996.
9. Toretsky, JA, and Helman, L. Cytokine Regulators of Ewing’s Family of Tumors Growth:
Is there an Autocrine Loop? American Society of Pediatric Hematology/Oncology, San
Francisco, CA, September 1997.
10. Wang, S., Zou, T-T., Kong, D., Huang, J., Souza, R., Smolinski, K., Abraham, J., Meltzer,
S., Toretsky, J. Growth Inhibition and Apoptotic Cell Death in Colorectal Cancer Cells
Induced by Blocking IGFIR or its Signal Transduction. American Association for Cancer
Research, New Orleans, LA, April 1998.
11. Toretsky, JA, Helman, LJ, Steinberg, S, Parente, C, Counts, D, Piromis, B, Thakar, M,
Eskenazi, A, Wexler, L. IGF-I and Apoptosis in the Ewing’s Sarcoma Family of Tumors.
American Society of Clinical Oncology, Atlanta GA., May 1999.
12. Thakar, M., Gutkind, S., Eskenazi, A., Toretsky, JA. Akt enables Ewing’s Sarcoma
Family of Tumors to survive doxorubicin therapy. American Association of Cancer
Research, Philadelphia, PA., April 1999.
13. Thakar, M., Eskenazi, A., Toretsky, JA. Blockade of phosphoinositide 3-OH kinase
enhances doxorubicin in Ewing’s Sarcoma Family of Tumors. Society Internalionale
Oncologie Pediatric/American Society of Pediatric Hematology and Oncology, Montreal,
CA., September 1999.
14. Rapoport, A, Simons-Evelyn, M, Bailey-Dell, K, Toretsky, JA, Ross, D, Fenton, R,
Kalvakolanu, D. PBK/TOPK-A novel mitotic kinase is upregulated in Burkitt’s Lymphoma
and other Neoplastic cell lines. American Society of Hematology, December 2000.
Toretsky, page
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15. Toretsky, Jeffrey A; Uren, Aykut; Mullholland, Justin; Rao, Srinavas; Thakar, Manjusha;
Skervsky, Amy; Mease, Ronnie. Noninvasive in vivo cell death detection in Ewing's
Sarcoma. American Association for Cancer Research, New Orleans, LA, March 2001.
16. Toretsky, Jeffrey A; Uren, Aykut; Mullholland, Justin; Rao, Srinavas; Thakar, Manjusha;
Skervsky, Amy; Weinberg, Irving, Line, Bruce, Mease, Ronnie. Non-invasive Detection of
Ewing’s Sarcoma Cell Death In Vivo. American Society of Pediatric Hematology and
Oncology/Society for Pediatric Research, Baltimore, MD, April 2001.
17. J. Toretsky, M. Thakar, A. Uren, A. Rice, T. Cripe Transcriptional modeling of EWS/FLI1:
a key to unlocking function. Brisbane, Australia, October 2001.
18. Aykut Üren, Amir A. Azari, Cassandra Berrian, Vladimir Wolf, Jeffrey S. Rubin, Jeffrey A.
Toretsky. Wnt/Frizzled Signaling in Ewing's Sarcoma. Keystone Symposia, Taos, NM,
March 2002.
19. Aykut Uren, Chen-Chih J Sun, Michelle Vitolo, Yu-Feng Sun, Maria Tsokos, Antonino
Passaniti, Jeffrey A Toretsky. Expression of Functional Beta-PDGF Receptors by
Ewing's Sarcoma. American Association for Cancer Research, San Francisco, CA, April
2002.
20. R.C. Mease*, I.N. Weinberg, J. Toretsky, J.F. Tait. Preparation of F-18 Labeled Annexin
V: A Potential Pet Radiopharmaceutical For Imaging Cell Death. J. Nucl. Med. 44:295P
Society for Nuclear Medicine, New Orleans, LA, June 2003.
21. J. Kitlinska, J. Toretsky, Z. Zukowska. Neuropeptide Y (NPY) and dipeptidyl peptidase
IV (DPPIV) inhibitor decrease Ewing’s sarcoma growth and enhance apoptosis.
American Society for Cell Biology, San Francisco, CA, December 2003.
22. Ogan D. Abaan, Amy Levenson, Aykut Uren, Jeffrey A. Toretsky. The protein tyrosine
phosphatase PTPL1 modulates Ewing’s sarcoma tumorigenesis. American Association
for Cancer Research, Orlando, FL, March 2004.
23. J. Kitlinska, J. Toretsky, Z. Zukowska. NPY and Growth of Neural-Crest Derived Tumors:
Differential Proliferative Effects and Angiogenesis. 7th International NPY Meeting,
Coimbra, Portugal, 2004.
24. Jeffrey A. Toretsky, Ogan D. Abaan, Amy Levenson, Aykut Uren. The protein tyrosine
phosphatase PTPL1 modulates Ewing’s sarcoma tumorigenesis. Protein Phosphatases.
FASEB Meeting, Snomass, CO, 2004.
25. Kitlinska J., Toretsky J., Pons J., Zukowska Z.: Proliferative effects of neuropeptide Y
(NPY) on neuroblastoma cells: synergistic interactions of multiple receptors. Experimental
Biology 2004, Washington, DC.
26. Kitlinska, J., Abe K., Kuo L., Pons, J., Yu, M., Li, L., Tilan J., Everhart L., Lee, E.W.,
Toretsky, J.A., Zukowska Z.. Role of neuropeptide Y and dipeptidyl peptidase IV in
regulation of growth of Ewing’s sarcoma family of tumors. Oral presentation at the 2nd
International Conference on Dipeptidyl Aminopeptidases, Magdeburg, Germany, 2005.
27. Kitlinska, J., Abe K., Kuo L., Pons, J., Yu, M., Li, L., Tilan, J., Zukowska, Z., Toretsky, J.
Neuropeptide Y in neural crest-derived pediatric tumors – effect on growth and
vascularization. Pediatric Academic Societies’ Annual Meeting, Washington, DC, May
2005.
28. Ogan D. Abaan, Judy Quong, Andrew Quong, Aykut Üren, Jeffrey A. Toretsky. PTPL1 is
a Transcriptional Target of the Oncogenic Fusion Protein EWS-FLI1 And May Modulate
IGF-IR Signaling. Protein Phosphorylation & Cell Signaling, Cold Spring Harbor, NY,
2005.
29. Ogan D. Abaan, Amy Levenson, Aykut Üren, Jeffrey A. Toretsky. The Protein Tyrosine
Phosphatase PTPL1 Modulates Ewing’s Sarcoma Tumorigenesis. Pediatric Academic
Societies Annual Meeting, Washington, DC, 2005.
Toretsky, page
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30. Hayriye Verda Erkizan, Ogan D. Abaan, Aykut Uren, Jeffrey A. Toretsky. RNA Helicase
A As A Partner Of EWS-FLI1 Protein In Ewing’s Sarcoma Cell Lines. PAS Meeting,
Washington DC, 2005.
31. Jeffrey A. Toretsky, Hayriye Erkizan, Ogan D. Abaan, Amy Levenson, Aykut Uren. RNA
Helicase A Is A Functional Partner Of EWS-FLI1 In Ewing’s Sarcoma Family Of Tumors.
Helicases And NTP Driven Nucleic Acid Machines, Arolla Switzerland 2005.
32. Laura Kelsey McCarty, Sunil Mandal, Alan Kozikowski, Jeffrey Toretsky. Select
phosphoinositol analogs reduce Ewing's sarcoma and leiomyosarcoma cell line growth.
AACR Molecular Targets and Cancer Therapeutics Meeting, Philadelphia, PA, 2005.
33. Hayriye Verda Erkizan, Ogan D. Abaan, Aykut Üren, Jeffrey A. Toretsky. RNA Helicase
A (RHA) Is A Functional Partner Of EWS-FLI1 In Ewing’s Sarcoma Family Of Tumors.
AACR Meeting Washington DC, 2006.
34. Shannon Fallen, Richard Schlegel, Elaine Fuchs, Jeffrey A. Toretsky, Aykut Üren.
Transgenic Mouse Models of Skin and Cervical Tumor Development Involving Betacatenin. Keystone Symposia, Wnt Meeting, Snowbird, Utah, 2006.
35. Jeffrey A. Toretsky, Verda Erkizan, Ogan D. Abaan, and Aykut Üren. Ewing sarcoma
oncoprotein EWS-FLI1 activity is enhanced by RNA helicase a (RHA). 31st FEBS
Congress, Molecules in Health and Disease, Istanbul, Turkey, 2006.
36. Kitlinska, J., Kuo, L., Tilan, J., Abe, K., Everhart, L., Li, L., Toretsky, J., Zukowska, Z.
Neuropeptide Y in neuroblastoma – effect on growth and vascularization. Oral
presentation at 8th NPY Meeting April 22-26, 2006 St. Petersburg, FL.
37. Kitlinska, J., Kuo, L., Tilan, J., Abe, K., Everhart, L., Li, L., Sun, C., Zukowska, Z. and
Toretsky, J., “Neuropeptide Y – a novel growth factor for neuroblastoma.” Advances in
Neuroblastoma Research Meeting, Los Angeles, CA, 2006.
38. Ogan D. Abaan, Sarah Pickard, Aykut Üren, Jeffrey A. Toretsky. Ikba May Be A
Physiological Target Of Protein Tyrosine Phosphatase L1 (PTPL1) In Ewing’s Sarcoma.
Protein Phosphatases. FASEB Meeting, Snowmass, CO, 2006.
39. Hayriye Verda Erkizan, Silke Schlottmann, L. Kelsey Mccarty, Shannon Fallen, Ogan D.
Abaan, Aykut Üren, Jeffrey A. Toretsky. RNA Helicase A (RHA) Enhances The
Oncogenic Function Of EWS-FLI1 In Ewing’s Sarcoma. Symposium On Biological
Complexity: “Disease Of Transcription", Salk Institute, San Diego, California, January 1114, 2007.
40. Endo Y, Beauchamp EM, Toretsky JA, Üren A, Rubin JS. Wnt-3a stimulates neurite
outgrowth in Ewing sarcoma cells. WNT 2007 Conference, University of California San
Diego, La Jolla, California, June 21-23, 2007
41. L. Kelsey McCarty, Pavel A. Petukhov, Sunil K. Mandal, Silke Schlottmann, Verda
Erkizan, Aykut Uren, Alan P. Kozikowski, Jeffrey A. Toretsky. Novel small molecule
inhibitors of Akt reduce Ewing's sarcoma cell growth. ASPHO Meeting, Toronto,
CANADA, 2007.
42. Jeffrey Toretsky, Hayriye Verda Erkizan, Silke Schlottmann, Shannon Fallon, Ogan D.
Abaan, MIlton Brown, Aykut Uren. Therapeutic targeting of EWS-FLI1: Inhibition of
Ewing’s Sarcoma growth using peptides and small molecule lead compounds. Connective
Tissue Oncology Society, Seattle, Washington, November 1-3, 2007. WON BEST
POSTER AWARD.
43. Chen K, Fallen S, Abaan HO, Toretsky JA, Üren A. Wnt10b Induces chemotaxis of
osteosarcoma and correlates with reduced survival. 13th annual Connective Tissue
Oncology Society (CTOS) Meeting, Seattle, Washington, November 1-3, 2007.
Toretsky, page
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44. Chen K, Fallen S, Abaan HO, Toretsky JA, Üren A. Wnt10b induces chemotaxis of
Osteosarcoma and correlates with reduced survival. Wnt/beta-Catenin Signaling.
Keystone, Colorado. Feb 17 - Feb 22, 2008.
45. Fallen S, Bulut G, Toretsky J, Üren A. Beta-catenin enhances HPV-E7 induced
tumorigenesis.Signaling Pathways in Cancer and Development. Steamboat Springs,
Colorado. March 24 - 29, 2008.
46. Bulut G, Beauchamp EM, Fallen S, Chen K, Toretsky JA, Khanna C, Üren A.
Molecular targeting of ezrin as an anti-metastatic agent. TASSA Annual conference,
Harvard University, Boston, Massachusetts. April 11-13, 2008
47. Hayriye Verda Erkizan, Silke Schlottmann, Shannon Fallon, Ogan D. Abaan, MIlton
Brown, Aykut Uren, Jeffrey Toretsky. Inhibition of Ewing’s Sarcoma growth using
peptides and small molecule lead compounds. American Association of Cancer
Research, San Diego, CA, April 2008. ORAL PRESENTATION.
48. Yali Kong, Julie S. Barber, Hayriye V. Erkizan, Melinda Merchant, Tsu-hang Chou,
Jeffrey A. Toretsky, Milton L. Brown. Synthesis and optimization of small molecule
inhibitors of EWS-FLI1. 236th ACS National Meeting, Philadelphia, PA, United States,
August 17-21, 2008.
49. Jeffrey A Toretsky, Hayriye Verda Erkizan, Silke Schlottmann, Yali Kong, Melinda
Merchant, Milton Brown, Aykut Uren Inhibition of Ewing’s Sarcoma Growth Via
Therapeutic Targeting of EWS-FLI1. National Academy Keck Futures Initiative, Irvine, CA
November 2008.
50. Toretsky JA, Erkizan HV, Schlottmann S, Kong Y, Merchant M, Brown M, Üren A.
Inhibition of Ewing’s Sarcoma Growth Using Therapeutic Targeting of EWS-FLI1. AACR,
Translational Cancer Medicine 2008: Bridging the Lab and the Clinic in Cancer Medicine,
November 3-6, 2008, Jerusalem, Israel.
51. Bulut G, Chen K, Glasgow E, Hong HS, Lee HS, Toretsky JA, Daar I, Khanna C, Üren A.
Small Molecule Inhibitors of Ezrin as Anti-Metastatic Agents in Osteosarcoma. 100th
AACR Annual Meeting, Denver, CO, April 17-22, 2009
52. Bulut G, Fallen S, Sun J, Kallakury B, Toretsky JA, Schlegel R, Üren A. Beta-catenin
Accelerates Human Papilloma Virus Type-16 Mediated Cervical Carcinogenesis WNT
2009 Meeting, Georgetown University, Washington DC, June 11-14, 2009
53. J Toretsky; H Verda Erkizan; Y Kong; M Merchant; J Barber-Rotenberg; M Brown; A
Uren. Therapeutic Targeting of EWS-FLI1: Small Molecule Protein-Protein Interaction
Inhibitors Prevent Xenograft Growth. Connective Tissue Oncology Society, Miami, FL,
11/6/09 (Oral Presentation).
54. G Bulut; K Chen; E Glasgow; S Hong; H Lee; J Toretsky; I Daar; C Khanna; A Uren
Small molecule inhibitors of ezrin as anti-metastatic agents in osteosarcoma. Connective
Tissue Oncology Society, Miami, FL, 11/7/09.
55. Barber-Rotenberg JS, Kong Y, Schnure N, Dakshanamurthy S, Frazier P, Erkizan HV,
Brown ML, Uren A, and Toretsky JA. EWS-FLI1 as a molecular target: Identification of
inhibitors for a disordered protein. AACR-NCI-EORTC Molecular Targets and Cancer
Therapeutics Meeting, Boston, MA. November 15-19, 2009.
56. Julie S. Barber-Rotenberg, Yali Kong, Nilan Schnure, Sivanesan Dakshanamurthy, Peter
Frazier, Hayriye V. Erkizan, Milton L. Brown, Aykut Uren, Jeffrey A. Toretsky. EWS-FLI1
as a molecular target: Small molecule inhibitors for a disordered protein. American
Association of Cancer Research, Washington, DC, April 2010.
57. Elspeth M. Beauchamp, Olga Rodriguez, Christopher Albanese, Jeffrey A. Toretsky,
and Aykut Üren. Arsenic Trioxide inhibits Ewing Sarcoma growth by blocking
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58.
59.
60.
61.
62.
63.
64.
65.
66.
67.
68.
69.
70.
Hedgehog/GLI pathway. American Association of Cancer Research, Washington, DC,
April 2010.
Gulay Bulut, Kevin Chen, Eric Glasgow, Sung-Hyeok Hong, Hyun-Shik Lee, Jeffrey A.
Toretsky, Ira Daar, Chand Khanna, Aykut Uren. Small molecule inhibitors of ezrin as
anti-metastatic agents in osteosarcoma. American Association of Cancer Research,
Washington, DC, April 2010.
Lauren M. Jacobwitz, Hayriye V. Erkizan, Aykut Üren, Jeffrey A. Toretsky. Phage
display identifies novel toxic binding peptides for the disordered oncoprotein EWSFLI1. American Association of Cancer Research, Washington, DC, April 2010.
Silke Schlottmann, Hayriye Verda Erkizan, Julie Barber-Rotenberg, Aykut Uren, MariaLaura Avantaggiati, Jeffrey A.Toretsky. EWS-FLI1 is regulated by acetylation. American
Association of Cancer Research, Washington, DC, April 2010.
Jeffrey A. Toretsky, Hayriye V. Erkizan, Yali Kong, Melinda Merchant, Julie S. BarberRotenberg, Milton L. Brown, Aykut Üren. Targeting of EWS-FLI1 with small molecule YK4-279 reduces xenograft growth by disruption of disordered protein-protein
interactions. American Association of Cancer Research, Washington, DC, April 2010.
Anna Kuan-Celarier, Congyi Lu, Lindsay Everhart, Jeffrey A. Toretsky, Joanna Kitlinska.
Neuropeptide Y in neuroblastoma – interactions with BDNF and effect on cell survival.
Advances in Neuroblastoma Research Association, June 2010.
Barber-Rotenberg JS, Kong Y, Tosso PN, Dakshanamurthy S, Erkizan HV, Brown ML,
Uren A, Toretsky JA. EWS-FLI1 as a Molecular Target: Inhibitors for a Disordered
Protein. Gordon Research Conference: Intrinsically Disordered Proteins. Davidson,
North Carolina, July 11-16, 2010.
Jeffrey A Toretsky, Hayriye Verda Erkızan, Yali Kong, Melinda Merchant, Vladimir N.
Uversky, Julie S. Barber-Rotenberg, Silke Schlottmann, Milton Brown, Aykut Üren.
Targeting of EWS-FLI1 with small molecule YK-4-279 reduces xenograft growth by
disruption of disordered protein-protein interactions Gordon Research Conference:
Intrinsically Disordered Proteins, Davidson, North Carolina, July 11-16, 2010.
Hayriye V. Erkizan, Lauren M. Scher, S. Ellen Gamble, Julie Barber-Rotenberg, Aykut
Üren, Jeffrey A Toretsky. Novel peptide directed targeting of EWS-FLI1 impairs tumor
cell growth. American Association of Cancer Research, Orlando, FL, April 2011.
Said Rahim, Elspeth M. Beauchamp, Yali Kong, Milton L. Brown, Jeffrey A. Toretsky,
Aykut Üren. YK4279 inhibits ERG and ETV1 mediated prostate cancer cell invasion.
American Association of Cancer Research, Orlando, FL, April 2011.
Gülay Bulut, Shannon Fallen, Elspeth M. Beauchamp, Lauren E. Drebing, Junfeng Sun,
Deborah L. Berry, Bhaskar Kallakury, Christopher P. Crum, Jeffrey A. Toretsky, Richard
Schlegel, Aykut Üren. Beta-catenin accelerates human papillomavirus type16 -E7
mediated cervical carcinogenesis in transgenic mice. American Association of Cancer
Research, Orlando, FL, April 2011.
Gülay Bulut, Sung-Hyeok Hong, Kevin Chen, Said Rahim, George W. Kosturko, Elspeth
M. Beauchamp, Eric Glasgow, Jeffrey A. Toretsky, Chand Khanna, Aykut Uren.
Development of small molecules to target ezrin as anti-metastatic agents. American
Association of Cancer Research, Orlando, FL, April 2011.
Jeffrey A Toretsky, Hayriye Verda Erkizan, Julie Barber-Rotenberg, Yali Kong, Milton
Brown, Aykut Uren. YK-4-279 is a novel small molecule directly targets EWS-FLI1.
American Society of Pediatric Hematology/Oncology Annual Meeting, Baltimore, MD April
14-17, 2011 (oral presentation).
Jeffrey A Toretsky, Hayriye Verda Erkızan, Julie S. Barber-Rotenberg, Aykut Üren.
Recognizing Intrinsic Protein Disorder in Small Molecule Therapeutic Development.
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71.
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79.
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81.
82.
National Cancer Institute Translational Science Meeting, Washington, DC, July 28-29,
2011 (oral presentation).
Ryan Commins, Aykut Üren, Jeffrey Toretsky. Combinations of current chemotherapies
with YK-4-279 in Ewing sarcoma. Combined Musculoskeletal Tumor Society/Connective
Tissue Oncology Society Meeting, Chicago, IL, October 26-29, 2011 (oral presentation).
Hayriye V. Erkizan, Lauren M. Scher, S. Ellen Gamble, Julie Barber-Rotenberg, Aykut
Üren, Jeffrey Toretsky. Novel peptide directed targeting of EWS-FLI1 impairs tumor cell
growth. Combined Musculoskeletal Tumor Society/Connective Tissue Oncology Society
Meeting, Chicago, IL, October 26-29, 2011 (oral presentation).
Gulay Bulut, Sung-Hyeok Hong, Kevin Chen, Elspeth Beauchamp, Said Rahim, George
Kosturko, Eric Glasgow, Sivanesan Dakshanamurthy, Hyun-Shik Lee, Ira Daar, Jeffrey
A. Toretsky, Chand Khanna, Aykut Üren. Small molecule inhibitors of ezrin inhibit the
metastasis of Osteosarcoma cells. Combined Musculoskeletal Tumor Society/Connective
Tissue Oncology Society Meeting, Chicago, IL, October 26-29, 2011.
Jason U. Tilan, Congyi Lu, Joshua Patrick Earnest, Lindsay M. Everhart, Asim Shabir,
Alison O’Neill, Shuo Wang, Magdalena Czarnecka, Jeffrey A. Toretsky, Joanna
Kitlinska. Differential functions of neuropeptide Y in Ewing’s sarcoma - hypoxia as a
switch. American Association of Cancer Research, Chicago. IL, April 3, 2012.
Jared T. Murdoch, Sung-Hyeok Hong, Gulay Bulut, George W. Kosturko, Lauren E.
Drebing, Jeffrey A. Toretsky, Aykut Uren. Small molecule inhibitor of ezrin inhibits
metastasis in a transgenic mouse model of osteosarcoma. American Association of
Cancer Research, Chicago. IL, April 3, 2012.
George W. Kosturko, Gulay Bulut, Sung-Hyeok Hong, Veronica Rodriguez, Milton Brown,
Jeffrey Toretsky, Chand Khanna, Mikell Paige, Aykut Uren. Design, synthesis and
biological evaluation of 2nd generation ezrin inhibitors for metastatic osteosarcoma.
American Association of Cancer Research, Chicago. IL, April 3, 2012.
David Solomon, Taeyon Kim, Laura Diaz-Martinez, Joshlean Fair, Abdel Elkahloun, Brent
Harris, Jeffrey Toretsky, Steven Rosenberg, Neerav Shukla, Marc Ladanyi, Yardena
Samuels, C. David James, Hongtao Yu, Jung-Sik Kim, Todd Waldman. Mutational
inactivation of STAG2 causes aneuploidy in human cancer. American Association of
Cancer Research, Chicago. IL, April 3, 2012.
S. Peter Hong, S. Ellen Gamble, Sung-Hyeok Hong, Christina L. Kobs, Karen E. Elsass,
Natalie L. South, Philip J. Monroe, Aykut Üren, Jeffrey A. Toretsky. LB-316 Pharmacokinetic modeling optimizes direct inhibition of the ‘undruggable’ target EWSFLI1 of Ewing Sarcoma. American Association of Cancer Research, Chicago. IL, April 3,
2012.
Julie S. Barber-Rotenberg, Saravana P. Selvanathan, Hayriye V. Erkizan, Maksymilian
Chruszcz, Milton L. Brown, Aykut Üren, Jeffrey A. Toretsky. EWS-FLI1 as a Molecular
Target: Inhibitors for a Disordered Protein. Gordon Research Conference: Intrinsically
Disordered Proteins, Mt. Snow, VT, July 2012.
Shah, N., Graham G., Li X., Wang J., Khan J., Toretsky J.A. HOX gene and associated
long noncoding RNA expression correlates with neuroblastoma cell line phenotype and
response to 13-cis retinoic acid. Advances in Neuroblastoma Research 2012, Toronto,
Ontario. June 2012
Shah, N., Graham G., Li X., Wang J., Khan J., Toretsky J.A. PBX1 is a Putative
Favorable Prognostic Biomarker as a Modulator of Retinoid Response in Neuroblastoma.
ASPHO 26th Annual Meeting, Miami, FL. May 2013
Shah, N., Graham G., Li X., Wang J., Khan J., Toretsky J.A. PBX1 is a Putative
Favorable Prognostic Biomarker as a Modulator of Retinoid Response in Neuroblastoma.
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84.
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COG Fall Meeting, Young Investigator Symposium, Dallas, TX. November 2013
Shah, N., Selich-Anderson, JA, Graham G., Siddiqui H., Li X., Wang J., Khan J.,
Toretsky JA. PBX1 is a Strong Favorable Prognostic Biomarker in Low-Risk and HighRisk Neuroblastoma and is Critical to Retinoid-Mediated Differentiation. Advances in
Neuroblastoma Research 2014, Cologne, Germany. May 2014
S. Ellen Gamble, Sung-Hyeok Hong, S. Peter Hong, Steve Summer, Philip J. Monroe,
Aykut Üren, Jeffrey A. Toretsky. Small molecule YK-4-279 inhibits the ‘undruggable’
target EWS-FLI1 leading to xenograft tumor regression. America Society of Clinical
Oncology, Chicago, Ill. June 2013.
AACR 2013
ABRCMS 2013
CTOS 2013
AACR 2014
Jeffrey Toretsky, Saravana P. Selvanathan, Garrett T. Graham, Hayriye V. Erkizan, Uta
Dirksen, Thanemozhi G. Natarajan, Cathy H. Wu, Elizabeth R. Lawlor, Aykut Üren. EWSFLI1 is an oncogene in a network and functioning in alternative splicing. Gordon
Research Conference Post-transcriptional Regulation. Newport, RI, 2014.
AACR 2015 - Haydar, Yasemine, Tsion, Stefan
Stefan K. Zöllner, Ryan Commins, Sung H. Hong, Hayriye V. Erkizan, Jeffrey A.
Toretsky. EWS-FLI1 targeted small molecule YK-4-279 synergizes with vinca alkaloids
through double hit to mitotic machinery. AACR, Philadelphia, PA. April 2015.
Elaine Yee Lin Chung, Valdemar Priebe, Eugenio Gaudio, Ivo Kwee, Chiara Tarantelli,
Andrea Rinaldi, Laura Carrassa, Monica Testoni, Luciano Cascione, Massimo Broggini,
Jeffrey A. Toretsky, Emanuele Zucca, Francesco Bertoni. The small molecule YK-4-279
shows anti-lymphoma activity in pre-clinical models. AACR, Philadelphia, PA, April 2015.
RNA 2015 -JT
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